UTSW - Study Finder

UTSW NORC Pilot Spinal Cord Injury Dietary Program

Description:

The goal of this observational study is to learn about the effects of a 9-week dietician-guided program modified from the National Diabetic Prevention Program (modified DPP-diet) in people with spinal cord injury on body composition and insulin sensitivity. The main question it aims to answer is: Does 9 week modified DPP-diet reduce body fat percentage and insulin resistance? Participants will: Have 9 weeks of Telehealth visit with dietician certified in providing DPP. Visit the laboratory before, immediately and 9 weeks after completion of the modified DPP-diet. Share with the researcher on the perceived benefit and obstacles in implementing the modified DPP-diet as part of their daily activities.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, LUCY.CHRISTIE@UTSouthwestern.edu

Principal Investigator: Yi-Ting Tzen

Gender: ALL

Age: 18 Years to 65 Years old

Phase: NA

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT06924177

IRB Number: STU20250145

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Interventions: OTHER: Telehealth with dietician

Conditions: Obesity and Obesity-related Medical Conditions, Spinal Cord Injury, Chronic

Keywords: spinal cord injuries, obesity, insulin resistance

Sites: UT Southwestern

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Optical Coherence Tomography Angiography in Neurological Disease

Description:

Optical Coherence Tomography Angiography (OCTA) is a non-invasive tool that images the neurovascular structures of the eye by using near-infrared light. Previous literature has demonstrated the potential of OCTA as a screening tool in stroke, but its utility in other neurological illness such as intracranial hemorrhage is unclear. Hence, this pilot study will gather preliminary data to support future grant applications to investigate this area more fully by recruiting patients with neurological illness and healthy controls and comparing their OCTA imaging parameters.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, Emily.Melikman@UTSouthwestern.edu

Principal Investigator: Noah Jouett

Gender: ALL

Age: 18 Years to old

Phase:

Healthy Volunteers:

This study is also accepting healthy volunteers

System ID: NCT06797765

IRB Number: STU-2024-0876

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Interventions: DEVICE: OCTA

Conditions: Octa, Stroke, Subarachnoid Hemorrhage, Intracerebral Hemorrhage, Brain and Nervous System

Sites: UT Southwestern

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Effect of the Stellate Ganglion Block on the Retinal Microcirculation

Description:

Surges in the sympathetic nervous system occur at the ictus of a variety of neurological critical illnesses including intracranial hemorrhage and ischemic stroke. It is hypothesized that these exaggerated increases in sympathetic nervous activity produce maladaptations that promote secondary brain injury. One of these possible mechanisms include diffuse vasospasm that cause cerebral ischemia. Hence, methods to abrogate the sympathetic nervous system in this context are under active investigation. One possible method is the regional anesthesia technique of the stellate ganglion nerve block, which is ordinarily used for complex regional pain syndrome, but has been shown to reduce cerebral sympathetic activity and reduces vasospasm in patients with subarachnoid hemorrhage. However, its effect on the microcirculation is not clear. Hence, we propose to study patients receiving the stellate ganglion nerve block as part of their standard medical care and to image their retinal microcirculation before and after the procedure using Optical Coherence Tomography Angiography (OCTA).

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, Emily.Melikman@UTSouthwestern.edu

Principal Investigator: Noah Jouett

Gender: ALL

Age: 18 Years to old

Phase:

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT06797752

IRB Number: STU-2024-1027

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Interventions: DEVICE: OCTA Scan

Conditions: Octa, Severe Brain Injury, Subarachnoid Hemorrhage, Intracerebral Hemorrhage

Sites: UT Southwestern

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Study of the Clinical and Radiological Impact of Ravulizumab in People With Neuromyelitis Optica Spectrum Disorder (AMAZE)

Description:

This is an observational study to: * evaluate the on-treatment clinical performance of ravulizumab in relation to the pre-treatment time period (time period prior to exposure), * enhance knowledge regarding conventional MRI outcomes in people with NMOSD treated with ravulizumab, * identify factors suggestive of subclinical disease progression through conventional MRI sequences, * determine if treatment with ravulizumab, impacts longitudinal 3D conformational MRI measures at the dorsal medulla and other regions of the CNS, and * identify biomarkers (e.g., serum neurofilament light chain (sNfL), conventional and novel MRI markers, etc.) related to disease activity.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, JOSE.SANTOYO@UTSouthwestern.edu

Principal Investigator: Darin Okuda

Gender: ALL

Age: 18 Years to old

Phase:

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT06398158

IRB Number: STU-2023-0744

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Interventions: DRUG: Ravulizumab

Conditions: Neuromyelitis Optica, Brain and Nervous System

Sites: UT Southwestern

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Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma

Description:

This phase III trial tests how well the addition of dinutuximab to Induction chemotherapy along with standard of care surgical resection of the primary tumor, radiation, stem cell transplantation, and immunotherapy works for treating children with newly diagnosed high-risk neuroblastoma. Dinutuximab is a monoclonal antibody that binds to a molecule called GD2, which is found on the surface of neuroblastoma cells, but is not present on many healthy or normal cells in the body. When dinutuximab binds to the neuroblastoma cells, it helps signal the immune system to kill the tumor cells. This helps the cells of the immune system kill the cancer cells, this is a type of immunotherapy. When chemotherapy and immunotherapy are given together, during the same treatment cycle, it is called chemoimmunotherapy. This clinical trial randomly assigns patients to receive either standard chemotherapy and surgery or chemoimmunotherapy (chemotherapy plus dinutuximab) and surgery during Induction therapy. Chemotherapy drugs administered during Induction include, cyclophosphamide, topotecan, cisplatin, etoposide, vincristine, and doxorubicin. These drugs work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing or by stopping them from spreading. Upon completion of 5 cycles of Induction therapy, a disease evaluation is completed to determine how well the treatment worked. If the tumor responds to therapy, patients receive a tandem transplantation with stem cell rescue. If the tumor has little improvement or worsens, patients receive chemoimmunotherapy on Extended Induction. During Extended Induction, dinutuximab is given with irinotecan, temozolomide. Patients with a good response to therapy move on to Consolidation therapy, when very high doses of chemotherapy are given at two separate points to kill any remaining cancer cells. Following, transplant, radiation therapy is given to the site where the cancer originated (primary site) and to any other areas that are still active at the end of Induction. The final stage of therapy is Post-Consolidation. During Post-Consolidation, dinutuximab is given with isotretinoin, with the goal of maintaining the response achieved with the previous therapy. Adding dinutuximab to Induction chemotherapy along with standard of care surgical resection of the primary tumor, radiation, stem cell transplantation, and immunotherapy may be better at treating children with newly diagnosed high-risk neuroblastoma.

Contact(s):

Call 833-722-6237
canceranswerline@utsouthwestern.edu

Principal Investigator: Tanya Watt

Gender: ALL

Age: to 30 Years old

Phase: PHASE3

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT06172296

IRB Number: STU-2024-0471

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Inclusion Criteria:

* Patients must be enrolled on APEC14B1 and have consented to testing through the Molecular Characterization Initiative (MCI), prior to enrollment on ANBL2131 * ≤ 30 years at the time of initial diagnosis with high-risk disease * \* Must have a diagnosis of neuroblastoma (NBL) or ganglioneuroblastoma (nodular) verified by tumor pathology analysis or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamines * Newly diagnosed, high risk neuroblastoma (HRNBL) defined as one of the following: * Any age with International Neuroblastoma Risk Group (INRG) Stage L2, MS, or M and MYCN amplification * Age ≥ 547 days and INRG stage M regardless of biologic features (clinical MYCN testing not required prior to enrollment) * Any age initially diagnosed with INRG Stage L1 MYCN amplified NBL who have progressed to stage M without systemic chemotherapy * Age ≥ 547 days of age initially diagnosed with INRG Stage L1, L2, or MS who have progressed to stage M without systemic chemotherapy (clinical MYCN testing not required prior to enrollment) * Patients must have a body surface area (BSA) ≥ 0.25 m\^2 * No prior anti-cancer therapy except as outlined below: * Patients initially recognized to have high-risk disease treated with topotecan/cyclophosphamide initiated on an emergent basis and within allowed timing, and with consent * Patients observed or treated with a single cycle of chemotherapy per a low or intermediate risk neuroblastoma regimen (e.g., as per ANBL0531, ANBL1232 or similar) for what initially appeared to be non-high-risk disease but subsequently found to meet the criteria * Patients who received localized emergency radiation to sites of life threatening or function-threatening disease prior to or immediately after establishment of the definitive diagnosis * Human immunodeficiency virus (HIV) -infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial * A serum creatinine based on age/sex as follows: * 1 month to \< 6 months: Male 0.4 mg/dL and female 0.4mg/dL * 6 months to \< 1 year: Male 0.5 mg/dL and female 0.5 mg/dL * 1 to \< 2 years: Male 0.6 mg/dL and female 0.6 mg/dL * 2 to \< 6 years: Male 0.8 mg/dL and female 0.8 mg/dL * 6 to \< 10 years: Male 1 mg/dL and female 1 mg/dL * 10 to \< 13 years: Male 1.2 mg/dL and female 1.2 mg/dL * 13 to \< 16 years: Male 1.5 mg/dL and female 1.4 mg/dL * ≥ 16 years: Male 1.7 mg/dL and female 1.4 mg/dL * The threshold creatinine values were derived from the Schwartz formula for estimating glomerular filtration rate (GFR) utilizing child length and stature data published by the Centers for Disease Control (CDC) * or a 24-hour urine creatinine clearance ≥ 70 mL/min/1.73 m\^2 or * or a GFR ≥ 70 mL/min/1.73 m\^2. GFR must be performed using direct measurement with a nuclear blood sampling method or direct small molecule clearance method (iothalamate or other molecule per institutional standard) * Note: Estimated GFR (eGFR) from serum creatinine, cystatin C or other estimates are not acceptable for determining eligibility * Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age * Serum glutamic pyruvic transaminase (SGPT) (Alanine aminotransferase \[ALT\]) ≤ 10 x ULN\* * Note: For the purpose of this study, the ULN for SGPT (ALT) has been set to the value of 45 U/L * \* Shortening fraction of ≥ 27% by echocardiogram, or * Ejection fraction of ≥ 50% by echocardiogram or radionuclide angiogram * Ability to tolerate Peripheral Blood Stem Cell (PBSC) collection: No known contraindication to PBSC collection. Examples of contraindications might be a weight or size less than the collecting institution finds feasible, or a physical condition that would limit the ability of the child to undergo apheresis catheter placement (if necessary) and/or the apheresis procedure

Exclusion Criteria:

* Patients who are 365-546 days of age with INRG Stage M and MYCN non-amplified NBL, irrespective of additional biologic features * Patients ≥ 547 days of age with INRG Stage L2, MYCN non-amplified NBL, regardless of additional biologic features * Patients with known bone marrow failure syndromes * Patients on chronic immunosuppressive medications (e.g., tacrolimus, cyclosporine, corticosteroids) for reasons other than prevention/treatment of allergic reactions and adrenal replacement therapy are not eligible. Topical and inhaled corticosteroids are acceptable * Patients with a primary immunodeficiency syndrome who require ongoing immune globulin replacement therapy * Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required prior to enrollment for female patients of childbearing potential * Lactating females who plan to breastfeed their infants * Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation * All patients and/or their parents or legal guardians must sign a written informed consent * All institutional, food and drug administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met

Interventions: PROCEDURE: Biospecimen Collection, PROCEDURE: Bone Marrow Aspiration, PROCEDURE: Bone Marrow Biopsy, DRUG: Carboplatin, DRUG: Cisplatin, PROCEDURE: Computed Tomography, DRUG: Cyclophosphamide, BIOLOGICAL: Dinutuximab, DRUG: Doxorubicin, PROCEDURE: Echocardiography Test, DRUG: Etoposide, PROCEDURE: FDG-Positron Emission Tomography and Computed Tomography Scan, PROCEDURE: Hematopoietic Cell Transplantation, DRUG: Irinotecan, DRUG: Isotretinoin, PROCEDURE: Leukapheresis, PROCEDURE: Magnetic Resonance Imaging, DRUG: Melphalan, PROCEDURE: Multigated Acquisition Scan, RADIATION: Radiation Therapy, PROCEDURE: Radionuclide Imaging, OTHER: Survey Administration, DRUG: Temozolomide, DRUG: Thiotepa, DRUG: Topotecan, PROCEDURE: Tumor Resection, DRUG: Vincristine

Conditions: Ganglioneuroblastoma, Nodular, Neuroblastoma, Brain and Nervous System

Sites: Children’s Health

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A Study to Estimate How Often Post-stroke Spasticity Occurs and to Provide a Standard Guideline on the Best Way to Monitor Its Development (EPITOME)

Description:

This study will monitor patients during the first year following their stroke. Stroke is a very serious condition where there is a sudden interruption of blood flow in the brain. The main aim of the study will be to find out how many of those who experience their first-ever stroke then go on to develop spasticity that would benefit from treatment with medication. Spasticity is a common post-stroke condition that causes stiff or ridged muscles. The results of this study will provide a standard guideline on the best way to monitor the development of post-stroke spasticity.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, Victoria.Castillo@UTSouthwestern.edu

Principal Investigator: Fatma Gul

Gender: ALL

Age: 18 Years to 90 Years old

Phase:

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT06055725

IRB Number: STU-2023-1049

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Conditions: Spasticity as Sequela of Stroke

Sites: UT Southwestern

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A Dose Escalation and Dose Expansion Study of Intratumoral ONM-501 Alone and in Combination With Cemiplimab in Patients With Advanced Solid Tumors and Lymphomas. (ON-5001)

Description:

A phase 1, multicenter, open label, non-randomized dose escalation and dose expansion study to examine the maximum tolerated dose, (MTD), minimum effective dose (MED) and/or recommended dose for expansion (RDE) of intratumoral ONM-501 as monotherapy and in combination with a PD-1 checkpoint inhibitor in patients with advanced solid tumors and lymphomas.

Contact(s):

Call 833-722-6237
canceranswerline@utsouthwestern.edu

Principal Investigator: Heather McArthur

Gender: ALL

Age: 18 Years and over

Phase: PHASE1

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT06022029

IRB Number: STU-2023-0921

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Inclusion Criteria:

• Ability to understand and willingness to sign written informed consent before performance of any study procedures
• Age ≥ 18 years
• Participants with solid tumors or lymphomas, confirmed by available histopathology records or current biopsy, that are advanced, nonresectable, or recurrent and progressing since last antitumor therapy, and for which no alternative standard therapy exists.
• Participants must have a minimum of one injectable and measurable lesion.
• Participants with prior Hepatitis B or C are eligible if they have adequate liver function
• Participants with human immunodeficiency virus (HIV) are eligible if on established HAART for a minimum of 4 weeks prior to enrollment, have an HIV viral load \<400 copies/mL, and have CD4+ T-cell (CD4+) counts ≥ 350 cells/uL
• Adequate bone marrow function:
• Adequate liver function

Exclusion Criteria:

Patients will be excluded from this study if they meet any of the following criteria (Part 1a and Part 1b).
• Other malignancy active within the previous 2 years except for basal or squamous cell skin cancer, superficial bladder cancer, or carcinoma in situ of the cervix or breast that has completed curative therapy.
• Major surgery within 4 weeks before the first dose of study drug.
• Brain metastases that are untreated or in the posterior fossa or involve the meninges. Participants with stable or previously treated progressing brain metastases (except in the posterior fossa or involving the meninges) may be permitted in a case-by-case basis at the Sponsor's discretion.
• Prolongation of corrected QT (QTc) interval to \>470 millisecond (ms) for males and females when electrolytes balance is normal.
• Females who are breastfeeding or pregnant at screening or baseline
• Females of childbearing potential that refuse to use a highly effective method of contraception.
• Has uncontrolled or poorly controlled hypertension as defined by a sustained BP \> 9. Has received prior investigational therapy within 5 half-lives of the agent or 4 weeks before the first administration of study drug, whichever is shorter.
• Has had any major cardiovascular event within 6 months prior to study drug 10. Has known hypersensitivity to any component in the formulation of ONM-501
• Has an active infection requiring systemic treatment
• Is participating in another therapeutic clinical trial Additional Exclusion Criteria for ONM-501 in Combination with cemiplimab (Part 1b)
• Has known hypersensitivity to any component in the formulation of cemiplimab
• Has any active or recent history of a known or suspected autoimmune disease or recent history of a syndrome that required systemic corticosteroids (\>10 mg daily prednisone equivalent)
• Has a condition requiring systemic treatment with corticosteroids

Interventions: DRUG: ONM-501, DRUG: Cemiplimab

Conditions: Triple Negative Breast Cancer, Diffuse Large B Cell Lymphoma, Follicular Lymphoma, Lymphoma, Non-Hodgkin, Mantle Cell Lymphoma, Bladder Cancer, Uveal Melanoma, Recurrent, Cervix Cancer, Carcinoma In Situ, Head and Neck Squamous Cell Carcinoma, Skin Cancer, Metastatic Cancer, Tumor, Solid, Tumor Recurrence, Anus, Bones and Joints, Brain and Nervous System, Breast - Female, Breast - Male, Cervix, Colon, Corpus Uteri, Hodgkins Lymphoma, Kaposis sarcoma, Kidney, Lung/Thoracic, Lymphoid Leukemia, Melanoma, skin, Multiple Myeloma, Mycosis Fungoides, Non-Hodgkins Lymphoma, Other Digestive Organ, Other Endocrine System, Other Skin, Rectum, Small Intestine, Soft Tissue, Stomach, Urinary Bladder

Keywords: Solid tumors, Lymphoma, ONM-501, STING, Intra-tumoral, HNSCC, Breast Cancer, Melanoma, Skin Cancer, cemiplimab, Libtayo, DLBCL, bladder cancer, cervical cancer, metastases, immunotherapy, ICI, TNBC, Triple Negative, mTNBC, anti-PD-1 antibody, BRCA1, BRCA2, anti-PD-L1, uveal, NHL, Mantle Zone lymphoma, FL, stimulator of interferon genes

Sites: UT Southwestern

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Emotional Cognition: Establishing Constructs and Neural-Behavioral Mechanisms in Older Adults with Depression (ENSURE)

Description:

This is a cross-sectional pilot study designed to establish hot and cold cognitive functions and underlying neurocircuitry in older adults with MDD. The investigators will study 120 participants aged 21-80 years old with MDD. All participants will undergo clinical and neurocognitive assessment, and Magnetoencephalography (MEG)/Magnetic resonance imaging (MRI) procedures at one time point. The investigators will also enroll 120 demographically matched comparable, never-depressed healthy participants (controls) to establish cognitive benchmarks. Healthy controls will complete clinical and neurocognitive measures at one time point. To attain a balanced sample of adults across the lifespan, the investigators will enroll participants such that each age epoch (e.g., 21-30, 31-40, etc.) has a total of ten subjects (n=10) in both the healthy control cohort and depressed cohort.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, Aatika.Parwaiz@UTSouthwestern.edu

Principal Investigator: Shawn McClintock

Gender: ALL

Age: 21 Years to 80 Years old

Phase:

Healthy Volunteers:

This study is also accepting healthy volunteers

System ID: NCT05966532

IRB Number: STU-2021-1131

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Interventions: BEHAVIORAL: Hot Cognitive Task, BEHAVIORAL: Cold cognitive tasks, OTHER: Structural magnetic resonance imaging (sMRI), OTHER: Magnetoencephalography imaging (MEG), BEHAVIORAL: four self-report forms per the requirement of the NIH Common Data Elements project, BEHAVIORAL: Four self-report measures to assess interpersonal functioning, BEHAVIORAL: Clinical assessments, OTHER: ATHF

Conditions: Major Depressive Disorder (MDD), Healthy Adult Volunteer, Brain and Nervous System

Keywords: Emotional Cognition

Sites: UT Southwestern

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Sonocloud-9 in Association With Carboplatin Versus Standard-of-Care Chemotherapies (CCNU or TMZ) in Recurrent GBM (SONOBIRD)

Description:

The brain is protected from any toxic or inflammatory molecule by the blood-brain barrier (BBB). This physical barrier is located at the level of the blood vessel walls. Because of these barrier properties, the blood vessels are also impermeable to the passage of therapeutic molecules from the blood to the brain. The development of effective treatments against glioblastoma is thus limited due to the BBB that prevents most drugs injected in the bloodstream from getting into brain tissue where the tumour is seated. The SonoCloud-9 (SC9) is an investigational device using ultrasound technology and specially developed to open the BBB in the area of and surrounding the tumour. The transient opening of the BBB allows more drugs to reach the brain tumour tissue. Carboplatin is a chemotherapy that is approved to treat different cancer types alone or in combination with other drugs, and has been used in the treatment of glioblastoma. Despite its proven efficacy in the laboratory on glioblastoma cells, carboplatin does not readily cross the BBB in humans. A clinical trial has shown that in combination with the SonoCloud-9, more carboplatin can reach the brain tumour tissue. The objective of the proposed trial is to show that the association - carboplatin with the SonoCloud-9 - will increase efficacy of the drug in patients with recurrent glioblastoma.

Contact(s):

Call 833-722-6237
canceranswerline@utsouthwestern.edu

Principal Investigator: Toral Patel

Gender: ALL

Age: 18 Years to old

Phase: PHASE3

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05902169

IRB Number: STU-2023-1253

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Inclusion Criteria:

• Histologically proven glioblastoma (WHO criteria 2021), absence of IDH mutation demonstrated by negative IDH1 R132H staining on Immunohistochemistry.
• Patient must have received prior first line therapy that must have contained both:
• Prior surgery or biopsy and standard fractionated radiotherapy (1.8-2 Gy/fraction, \>56 Gy\<66 Gy) or hypofractionated radiotherapy (15 x 2.66 Gy or similar regimen)
• One line of maintenance chemotherapy and/or immune- or biological therapy, (with or without Tumor-Treating Fields)
• First, unequivocal disease progression with
• measurable tumor (\>100 mm2 or 1 cm3, based on RANO criteria) documented (e.g., increase of 25% in tumor diameter) on MRI performed within 14 days of inclusion and,
• interval of a minimum of 12 weeks since the completion of prior radiotherapy, unless there is a new lesion outside the radiation field or unequivocal evidence of viable tumor on histopathological sampling
• Patient is candidate for craniotomy and at least 50% resection of enhancing region
• Maximal enhancing tumor diameter prior to inclusion ≤ 5 cm on T1w. (In case of planned lobectomy, post operative peritumoral brain or residual size ≤5 cm)
• WHO performance status ≤ 2 (equivalent to Karnofsky Performance Status (KPS) ≥ 70)
• Age ≥ 18 years
• Participant must be recovered from acute toxic effects (\ • ≥ 4 weeks or 5 half-lives (whichever is shorter) for * Cytotoxic * Other small chemical entity (e.g., targeted therapy) * For biologics (e.g., antibodies, except bevacizumab)
• ≥ 6 weeks of prior bevacizumab
• Adequate hematologic, hepatic, and renal laboratory values within 14 days of inclusion i.e.:
• Hemoglobin ≥ 10 g/dL, platelets ≥ 100,000/mm3, neutrophils ≥ 1500/mm3.
• Liver function test with ≤ grade 1 alterations, except if due to antiepileptic drug therapy or isolated increased bilirubin due to Gilbert syndrome
• Estimated glomerular filtration rate (eGFR) of at least 60 mL/min/1.73 m2 using Cockcroft Gault formula
• Patient able to understand clinical trial information and willing to provide signed and informed consent
• Patient of childbearing potential must have a negative pregnancy test within 14 days of inclusion and must agree to use a medically-acceptable method of birth control during the treatment period and, if randomized in the experimental arm, for at least 1 month after the last cycle of carboplatin
• A male patient must agree to use condoms during the treatment period and, if randomized in the experimental arm, for at least 3 months after the last cycle of carboplatin; the patient must also refrain from donating sperm during this period.
• Patient must be a beneficiary of a health plan that covers routine patient care costs. Patient must be a beneficiary of or affiliated with a social security scheme (according to country-specific requirements) Non-

Inclusion Criteria:

• Multifocal enhancing tumor on T1w (unless all localized in a 5 cm diameter area)
• Posterior fossa tumor
• Known BRAF/ NTKR mutated patients
• Patient at risk of surgery site infection (e.g., 2 or more previous craniotomies/neurosurgery within the last 3 months, poor skin condition, and/or previously infected surgical field, or any other condition that is of increased infectious risk in the opinion of the neurosurgeon)
• Patient treated at high, stable -or average- dose of corticosteroids (≥ 6 mg/day dexamethasone or equivalent) in the 7 days prior to inclusion. Patients on dexamethasone for reasons other than mass effect may still be enrolled.
• Contra-indication to carboplatin, CCNU or TMZ
• Known history of hypersensitivity reactions to perflutren lipid microsphere components or to any of the inactive ingredients in ultrasound resonator
• Patient has received bevacizumab for other reasons (such as tumor progression) than treating edema
• Peripheral neuropathy or neuropathy ≥ grade 2
• Uncontrolled epilepsy or evidence of intracranial pressure
• Patient with known intracranial aneurism or having presented intra-tumor significant spontaneous hemorrhage
• Patient with unremovable coils, clips, shunts, intravascular stents, and/or wafer, or reservoirs
• Patient with medical need to be on continued anti-platelet aggregation therapy and/or anticoagulation. Patients for whom anticoagulation/platelet aggregation can be temporarily interrupted may be eligible after discussion and prior authorization by the sponsor.
• Patient receiving enzyme-inducing antiepileptic drugs (namely phenytoin, carbamazepine and derivatives, phenobarbital), unless switched on another antiepileptic regimen
• History of other malignancy within 3 years prior to study start with the exception of adequately treated basal cell carcinoma, squamous cell carcinoma, non-melanomatous skin cancer or carcinoma in situ of the uterine cervix
• Patient with known or suspected active or chronic infections
• Patient with known significant cardiac disease, known to have right-to-left shunts, severe pulmonary hypertension (pulmonary artery pressure \> 90 mm Hg), uncontrolled systemic hypertension, or acute respiratory distress syndrome
• Known sensitivity/allergy to gadolinium, or other intravascular contrast agents
• Patient with impaired thermo-regulation or temperature sensation
• Pregnant, or breastfeeding patient
• Any other serious patient medical or psychological condition that may interfere with adequate and safe delivery of treatment and care (e.g., positive human immunodeficiency virus \[HIV\] status, potential blood-borne infections,...), circumstance (e.g., sinus opening during surgery), psychological, morphological characteristics (e.g., skin characteristics, bone thickness), or any pre-existing comorbidities that in the investigator's opinion may prevent the implantation of the device, may impair the ability of the patient to receive treatment with SonoCloud-9 or may be confounding for evaluation of the clinical trial endpoints
• Patients under guardianship, curatorship, under legal protection or deprived of liberty by an administrative or judicial decision Exclusion Criterion: Occurrence of any major medical illnesses or impairments that in the Investigator's opinion may hampered the ability of the patient to receive treatment with SonoCloud-9 or may be confounding for evaluation of the clinical endpoints.

Interventions: DEVICE: SonoCloud-9 (SC9), DRUG: Carboplatin, DRUG: Lomustine, DRUG: Temozolomide

Conditions: Glioblastoma, Recurrent Glioblastoma, GBM, Brain and Nervous System

Keywords: carboplatin, SonoCloud, blood-brain barrier, Low Intensity Pulsed Ultrasound (LIPU)

Sites: UT Southwestern

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A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy (HINALEA 2)

Description:

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled are children \<2 years of age genetically diagnosed with SMA.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, Tammy.Ramm@UTSouthwestern.edu

Principal Investigator: Kaitlin Batley

Gender: ALL

Age: 3 Months to 24 Months old

Phase: PHASE4

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05861999

IRB Number: STU-2023-1071

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Interventions: DRUG: risdiplam

Conditions: Muscular Atrophy, Spinal

Sites: Children’s Health

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A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy (HINALEA 1)

Description:

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children \< 2 years of age genetically diagnosed with SMA.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, Tammy.Ramm@UTSouthwestern.edu

Principal Investigator: Kaitlin Batley

Gender: ALL

Age: 3 Months to 24 Months old

Phase: PHASE4

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05861986

IRB Number: STU-2023-0862

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Interventions: DRUG: risdiplam

Conditions: Muscular Atrophy, Spinal, Other

Sites: Children’s Health

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A Study With Tovorafenib (DAY101) as a Treatment Option for Progressive, Relapsed, or Refractory Langerhans Cell Histiocytosis

Description:

This phase II trial tests the safety, side effects, best dose and activity of tovorafenib (DAY101) in treating patients with Langerhans cell histiocytosis that is growing, spreading, or getting worse (progressive), has come back (relapsed) after previous treatment, or does not respond to therapy (refractory). Langerhans cell histiocytosis is a type of disease that occurs when the body makes too many immature Langerhans cells (a type of white blood cell). When these cells build up, they can form tumors in certain tissues and organs including bones, skin, lungs and pituitary gland and can damage them. This tumor is more common in children and young adults. DAY101 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Using DAY101 may be effective in treating patients with relapsed or refractory Langerhans cell histiocytosis.

Contact(s):

Call 833-722-6237
canceranswerline@utsouthwestern.edu

Principal Investigator: Erin Butler

Gender: ALL

Age: 180 Days to 22 Years old

Phase: PHASE2

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05828069

IRB Number: STU-2023-0818

Show full eligibility criteria

Inclusion Criteria:

* 180 days- \< 22 years (at time of study enrollment) * Patient must have a body surface area of ≥ 0.3 m\^2 * Patients with progressive, relapsed, or recurrent LCH with measurable disease at study entry * Patients must have had histologic verification of LCH (from either original diagnosis or relapse/progression) at the time of study entry (must be obtained within 28 days prior to enrollment and start of protocol therapy) (repeat if necessary) * Tissue confirmation of relapse is recommended but not required * Pathology report must be submitted for central confirmation of diagnosis within 7 days of enrollment. * Formalin-fixed paraffin-embedded (FFPE) blocks or unstained slides (initial diagnosis and/or subsequent biopsies) will be required for retrospective central confirmation of diagnosis and molecular studies * Patients with mixed histiocytic disorders (e.g. LCH with juvenile xanthogranuloma) may be included * Patients must have measurable disease, documented by radiographic imaging (LCH- specific response criteria (must be obtained within 28 days prior to enrollment and start of protocol therapy) (repeat if necessary). * Patients must have progressive or refractory disease or experience relapse after at least one previous systemic treatment strategy * Pathogenic somatic mutation detected in genes encoding tyrosine kinase receptors (CSFR1, ERBB3 or ALK), RAS or RAF (may be from original or subsequent biopsy or peripheral blood/bone marrow aspirate). Clinical mutation reports may include quantitative polymerase chain reaction (PCR) (e.g. BRAFV600E) and/or Sanger or next generation sequencing. Immunohistochemistry (e.g. VE1 antibody for BRAFV600E) alone is not sufficient * Participant must be able to take an enteral dose and formulation of medication. Study medication is only available as an oral suspension or tablet, which may be taken by mouth or other enteral route such as nasogastric, jejunostomy, or gastric tube * Karnofsky \>= 50% for patients \> 16 years of age and Lansky \>= 50% for patients =\< 16 years of age * Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1 or 2. Use Karnofsky for patients \> 16 years of age and Lansky for patients =\< 16 years of age * Myelosuppressive chemotherapy: Patients must not have received within 14 days of entry onto this study * Investigational agent or any other anticancer therapy not defined above: Patients must not have received any investigational agent or any other anticancer therapy (including MAPK pathway inhibitor) for at least 14 days prior to planned start of tovorafenib (DAY101) * Radiation therapy (RT): Patient must not have received RT within 2 weeks after the last dose fraction of RT * Patients must have fully recovered from any prior surgery * Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, targeted inhibitor, and/or radiotherapy with toxicities reduced to grade 1 or less (Common Terminology Criteria for Adverse Events \[CTCAE\] version 5.0) * Steroids: =\< 0.5 mg/kg/day of prednisone equivalent (maximum 20 mg/day) averaged during the month prior to study enrollment is permissible * Strong inducers or inhibitors of CYP2C8 are prohibited for 14 days before the first dose of tovorafenib (DAY101) and from planned administration for the duration of study participation * Medications that are breast cancer resistant protein (BCRP) substrates that have a narrow therapeutic index are prohibited for 14 days before the first dose of tovorafenib (DAY101) and for the duration of study participation * Peripheral absolute neutrophil count (ANC) \>= 750/uL unless secondary to bone marrow involvement, in such cases bone marrow involvement must be documented (must be performed within 7 days prior to enrollment, must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment) * Platelet count \>= 75,000/uL (unsupported/without transfusion within the past 7 days) (must be performed within 7 days prior to enrollment, must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment) * Patients with marrow disease must have platelet count of \>= 75,000/uL (transfusion support allowed) and must not be refractory to platelet transfusions. Bone marrow involvement must be documented * Hemoglobin \>= 8 g/dL (unsupported/without transfusion within the past 7 days). Patients with marrow disease must have hemoglobin \>= 8 g/dL (transfusion support allowed). Bone marrow involvement must be documented * Hematopoietic growth factors: At least 14 days after the last dose of a long-acting growth factor (e.g., Neulasta \[registered trademark\]) or 7 days for short-acting growth factor * A serum creatinine based on age/sex as follows (must be performed within 7 days prior to enrollment, must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment) * Age: 6 months to \< 1 year; Maximum Serum Creatinine (mg/dL):= 0.5 mg/dl (male and female) * Age: 1 to \< 2 years; Maximum Serum Creatinine (mg/dL): = 0.6 mg/dl (male and female) * Age: 2 to \< 6 years; Maximum Serum Creatinine (mg/dL): = 0.8 mg/dl (male and female) * Age: 6 to \< 10 years; Maximum Serum Creatinine (mg/dL): = 1.0 mg/dl (male and female) * Age: 10 to \< 13 years; Maximum Serum Creatinine (mg/dL): = 1.2 mg/dl (male and female) * 13 to \< 16 years; Maximum Serum Creatinine (mg/dL): = 1.5 mg/dl (male) and 1.4 mg/dl (female) * Age: \>= 16 years; Maximum Serum Creatinine (mg/dL): = 1.7 mg/dl (male) and 1.4 mg/dl (female) * OR- a 24 hour urine creatinine clearance \>= 50 mL/min/1.73 m\^2 * OR- a glomerular filtration rate (GFR) \>= 50 mL/min/1.73 m\^2. GFR must be performed using direct measurement with a nuclear blood sampling method OR direct small molecule clearance method (iothalamate or other molecule per institutional standard) * Note: Estimated GFR (eGFR) from serum creatinine, cystatin C or other estimates are not acceptable for determining eligibility * Bilirubin (sum of conjugated + unconjugated) =\< 1.5 x upper limit of normal (ULN) for age (must be performed within 7 days prior to enrollment, must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment) * Alanine aminotransferase (ALT) =\< 3 x ULN for age (must be performed within 7 days prior to enrollment, must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment) * Serum albumin \>= 2 g/dl must be performed within 7 days prior to enrollment, must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment) * For patients with liver disease caused by their histiocytic disorder (as evaluated on radiographic imaging or biopsy): patients may be enrolled with abnormal bilirubin, aspartate aminotransferase (AST), ALT and albumin with documentation of histiocytic liver disease * Fractional shortening (FS) of \>= 25% or ejection fraction of \>= 50%, as determined by echocardiography or multigated acquisition scan (MUGA) within 28 days prior to study enrollment. Depending on institutional standard, either FS or left ventricular ejection fraction (LVEF) is adequate for enrollment if only one value is measured; if both values are measured, then both values must meet criteria above (must be obtained within 28 days prior to enrollment and start of protocol therapy) (repeat if necessary) * No evidence of dyspnea at rest, no exercise intolerance, and a pulse oximetry \> 94% if there is clinical indication for determination; unless it is due to underlying pulmonary LCH * Central Nervous System Function Defined As: * Patients with seizure disorder may be enrolled if well controlled * Central nervous system (CNS) toxicity =\< Grade 2 * Human immunodeficiency virus (HIV) infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial unless antiretroviral therapy interacts with the metabolism of tovorafenib (DAY101) and cannot safely be changed to antivirals that do not interact with study medication

Exclusion Criteria:

* LCH arising along with other hematologic malignancy (e.g. mixed LCH with acute lymphoblastic leukemia) or any history of non-histiocytic malignancy * Disease scenarios as below will be excluded * Skin-limited disease * Gastrointestinal (GI) tract involvement only (those that have disease that can be determined by endoscopic biopsies only) * LCH-associated neurodegeneration (LCH-ND) without parenchymal lesions or other systemic lesions * Patients with activating mutations in MAP2K1 are not eligible for this study due to drug target specificity. Mutation status will be submitted to study team within 7 days of enrollment * Refractory nausea and vomiting, malabsorption, or external biliary shunt that would preclude adequate absorption of tovorafenib (DAY101) * Uncontrolled systemic bacterial, viral, or fungal infection * Major surgical procedure or significant traumatic injury within 14 days prior to study enrollment, or anticipation of need for major surgical procedure during the course of the study. Placement of a vascular access device or minor surgery is permitted within fourteen (14) days of study enrollment (provided that the wound has healed) * History of significant bowel resection that would preclude adequate absorption or other significant malabsorptive disease * Ophthalmologic considerations: Patients with known significant ophthalmologic conditions or known risk factors for retinal vein occlusion (RVO) or central serous retinopathy (CSR) are not eligible * History of solid organ or hematopoietic bone marrow transplantation * Clinically significant active cardiovascular disease, or history of myocardial infarction, or deep vein thrombosis/pulmonary embolism within 6 months prior to enrollment, ongoing cardiomyopathy, or current prolonged QT interval \> 440 ms based on triplicate electrocardiogram (ECG) average * History of Grade \>= 2 CNS hemorrhage or history of any CNS hemorrhage within 28 days of study entry * History of any drug reaction with eosinophilia and systemic symptoms (DRESS) syndrome or Stevens Johnsons syndrome (SJS) or who are allergic to tovorafenib (DAY101) or any of its components * CTCAE version (V). 5.0 Grade 3 symptomatic creatinine kinase (CPK) elevation ( \> 5 x ULN) * Female patients who are pregnant are ineligible. A pregnancy test is required for female patients of childbearing potential * Lactating females who plan to breastfeed their infants are ineligible * Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation are ineligible. Women of childbearing potential must use non-hormonal contraception during tovorafenib treatment and for at least 28 days after the last dose. Men should use effective contraception and must not father a child while taking tovorafenib and for 14 days after the last dose

Interventions: PROCEDURE: Biospecimen Collection, PROCEDURE: Bone Marrow Aspiration, PROCEDURE: Bone Marrow Biopsy, PROCEDURE: Computed Tomography, PROCEDURE: Echocardiography Test, PROCEDURE: FDG-Positron Emission Tomography and Computed Tomography Scan, PROCEDURE: Lumbar Puncture, PROCEDURE: Multigated Acquisition Scan, DRUG: Tovorafenib

Conditions: Recurrent Langerhans Cell Histiocytosis, Refractory Langerhans Cell Histiocytosis, Bones and Joints, Other Skin, Brain and Nervous System, Liver, Lung/Thoracic, Other Hematopoietic, Small Intestine

Sites: Children’s Health

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Phase 1/2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD) (FORTITUDE)

Description:

A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Adult Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, Jonathan.Thompson@UTSouthwestern.edu

Principal Investigator: Jaya Trivedi

Gender: All

Age: 18 Years to 65 Years old

Phase: Phase 1/Phase 2

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05747924

IRB Number: STU-2022-1177

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Interventions: Drug: AOC 1020, Drug: Placebo

Conditions: FSHD, FSHD1, FSHD2, FMD, FMD2, Fascioscapulohumeral Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy Type 1, Fascioscapulohumeral Muscular Dystrophy Type 2, Dystrophies, Facioscapulohumeral Muscular, Dystrophy, Facioscapulohumeral Muscular, Facioscapulohumeral Muscular Dystrophy 1, Facioscapulohumeral Muscular Dystrophy 2, Facio-Scapulo-Humeral Dystrophy, Atrophy, Facioscapulohumeral, Atrophies, Facioscapulohumeral, Facioscapulohumeral Atrophy, Muscular Dystrophies, Muscular Dystrophy, Facioscapulohumeral, FSH Muscular Dystrophy, Landouzy Dejerine Dystrophy, Landouzy-Dejerine Muscular Dystrophy, Dystrophies, Landouzy-Dejerine, Dystrophy, Landouzy-Dejerine, Landouzy-Dejerine Syndrome, Muscular Dystrophy, Landouzy Dejerine, Progressive Muscular Dystrophy, FSH

Keywords: FORTITUDE, Avidity, Avidity Biosciences, AOC 1020

Sites: UT Southwestern

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AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)

Description:

RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain. This is a multicenter, open-label dose evaluation clinical study to assess the safety, tolerability, and clinical efficacy of a one-time intravenous (IV) dose of RGX-202 in participants with Duchenne.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, Elaine.Most@UTSouthwestern.edu

Principal Investigator: Susan Iannaccone

Gender: MALE

Age: 1 Year to 11 Years old

Phase: PHASE2

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05693142

IRB Number: STU-2022-0270

Show full eligibility criteria

Part 1 - Key

Inclusion Criteria:

* The participant's legal guardian(s) is (are) willing and able to provide written, signed informed consent prior to any study-related procedures; and, where applicable, the minor participant has provided written or verbal assent according to local requirements. * Is a male at least 4 years of age and less than 12 years of age at consent or 1 to \<4 years of age at the time of dosing and ≥ 10 kg at the time of screening. * Must meet any of the following criteria: * DMD gene mutation in exons 18 and above, and a clinical picture consistent with typical DMD with the exception of a participant (Cohort 1b) with DMD gene mutation in exons 12-17. * Participant is able to walk 100 meters independently without assistive devices. Cohort 2c participant must be able to walk 10 meters independently without assistive devices. Cohort 1b participant must be able to walk with or without assistive devices. * Participant is able to complete the TTSTAND per protocol-specific criteria. * Participant has been on a stable dose of systemic glucocorticoids according to the standard of care for at least 12 weeks. Cohort 2c participants must be consistently on or off a stable dose of systemic glucocorticoids according to the standard of care for at least 12 weeks. * Clinical laboratory test results, including hepatic and renal function, are within the normal range during screening, or if abnormal, are not clinically significant, in the opinion of the investigator. * Documentation is provided at screening visit for participant's adherence to the local country's vaccination schedule. The parent(s) or legal guardian(s) must be willing to have their child receive a meningococcal vaccine, if not already vaccinated. * Participant and parent(s)/legal guardian(s) are willing and able to comply with scheduled visits, study intervention administration plan, and study procedures. Part 2 and 3

Inclusion Criteria:

* The participant's legal guardian(s) is (are) willing and able to provide written, signed informed consent prior to any study-related procedures; and, where applicable, the minor participant has provided written or verbal assent according to local requirements. * DMD gene mutation with any mutation except for those with deletions or point mutations in exons 8, 9 and/or 10. * Participant is able to complete the TTSTAND per protocol-specific criteria. * Clinical laboratory test results, including hepatic and renal function, are within the normal range during screening, or if abnormal, are not clinically significant, in the opinion of the investigator. * Documentation is provided at screening visit for participant's adherence to the local country's vaccination schedule. The parent(s) or legal guardian(s) must be willing to have their child receive a meningococcal vaccine, if not already vaccinated. * Participant and parent(s)/legal guardian(s) are willing and able to comply with scheduled visits, study intervention administration plan, and study procedures. * Is a male at least 1 year of age and ≥ 10 kg at the time of screening. * Participants 1 to \<4 years of age must meet the following criteria: * is able to walk 10 meters independently without assistive devices. * must be consistently on or off a stable dose of systemic glucocorticoids according to the standard of care for at least 12 weeks. * Participants 4 years and older must meet the following criteria: * are able to walk 100 meters independently without assistive devices. * have been on a stable dose of systemic glucocorticoids according to the standard of care for at least 12 weeks. * have a NSAA total score ≥16. Part 1

Exclusion Criteria:

* Participant has any condition that would contraindicate treatment with immunosuppression. * Participant has received ataluren (a protein restoration therapy) or an exon-skipping therapy for the treatment of DMD within 6 months of study entry or is unable to refrain from taking ataluren or exon-skipping therapy for a duration of 5 years from the time of RGX-202 administration. * Participant has received any investigational or commercial gene therapy product over his lifetime. * Participant is currently taking any other investigational intervention (other than corticosteroids) or has taken any other investigational intervention (other than corticosteroids) within 3 months prior to the scheduled Day 1 intervention. If your corticosteroid is vamorolone, the participant will be asked to temporarily convert his daily dosing to prednisolone/prednisone during a short period of time around RGX-202 administration. He will be allowed to revert back to his baseline vamorolone regimen at the original per kilogram dose at which he entered the study and should remain on this for 24 months unless the investigator determines that this is not clinically indicated or possible. * Participant has impaired cardiac function defined as a left ventricular ejection fraction of \< 55% on screening cardiac assessments (echocardiogram or MRI). * Participant is not a good candidate for the study, in the opinion of the investigator. Part 2 and 3

Exclusion Criteria:

* Participant has any condition that would contraindicate treatment with immunosuppression. * Participant has received givinostat within 3 months of study entry or has received ataluren (a protein restoration therapy) or an exon-skipping therapy for the treatment of DMD within 6 months of study entry or is unable to refrain from taking ataluren or exon-skipping therapy for a duration of 5 years from the time of RGX-202 administration. * Participant has received any investigational or commercial gene therapy product over his lifetime. * Participant is currently taking any other investigational intervention (other than corticosteroids) or has taken any other investigational intervention (other than corticosteroids) within 3 months prior to the scheduled Day 1 intervention. If your corticosteroid is vamorolone, the participant will be asked to temporarily convert his daily dosing to prednisolone/prednisone during a short period of time around RGX-202 administration. He will be allowed to revert back to his baseline vamorolone regimen at the original per kilogram dose at which he entered the study and should remain on this for 24 months unless the investigator determines that this is not clinically indicated or possible. * Participant has detectable AAV8 total binding antibodies in serum. * Participant has impaired cardiac function defined as a left ventricular ejection fraction of \< 55% on screening cardiac assessments echocardiogram or MRI). * Participant is not a good candidate for the study, in the opinion of the investigator.

Interventions: GENETIC: RGX-202

Conditions: Duchenne Muscular Dystrophy

Keywords: Gene therapy, DMD, Duchenne Muscular Dystrophy, Duchenne

Sites: Children’s Health

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Alzheimer's Disease Neuroimaging Initiative 4 (ADNI4)

Description:

Since its launch in 2004, the overarching aim of the Alzheimer's Disease Neuroimaging Initiative (ADNI) Study has been to validate biomarkers for Alzheimer's disease (AD) clinical trials. ADNI4 continues the previously funded ADNI1, ADNI-GO, ADNI2, and ADNI3 studies that have combined public/private collaborations between academia and industry to determine the relationships between the clinical, cognitive, imaging, genetic and biochemical biomarker characteristics of the entire spectrum of AD.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, Gisella.Rodriguez-Larrain@UTSouthwestern.edu

Principal Investigator: Trung Nguyen

Gender: ALL

Age: 55 Years to 90 Years old

Phase:

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05617014

IRB Number: STU-2023-0505

Show full eligibility criteria

Inclusion Criteria for Newly Enrolled Participants, CN Cohort:
• Participant may or may not have a significant subjective memory concern as reported by participant, study partner, or clinician.
• Normal memory function documented by scoring above demographically-adjusted cutoffs on the Logical Memory II subscale (Delayed Paragraph Recall, Paragraph A only) from the Wechsler Memory Scale - Revised (the maximum score is 25):
• ≥9 for 16 or more years of education
• ≥ 5 for 8-15 years of education
• ≥ 3 for 0-7 years of education
• Note: cut-offs may be modified over time as the field evolves in this area
• Mini-Mental State Exam score between 24 and 30 (inclusive) (Exceptions may be made for participants with less than 8 years of education at the discretion of the Project Director and/or Clinical Core)
• Clinical Dementia Rating = 0. Memory Box score must be 0.
• Cognitively normal, based on an absence of significant impairment in cognitive functions or activities of daily living.
• Stability of Permitted Medications for 4 weeks. In particular, participants may:
• Take stable doses of antidepressants lacking significant anticholinergic side effects (if they are not currently depressed and do not have a history of major depression within the past 1 years)
• Estrogen replacement therapy is permissible
• Gingko biloba is permissible, but discouraged
• Washout from psychoactive medication (e.g., excluded antidepressants, neuroleptics, chronic anxiolytics or sedative hypnotics, etc.) for at least 4 weeks prior to screening. Inclusion Criteria for Newly Enrolled Participants, MCI Cohort
• Participant must have a subjective memory concern as reported by participant, study partner, or clinician.
• Abnormal memory function documented by scoring within the demographically- adjusted ranges on the Logical Memory II subscale (Delayed Paragraph Recall, Paragraph A only) from the Wechsler Memory Scale - Revised (the maximum score is 25):
• ≤11 for 16 or more years of education
• ≤9 for 8-15 years of education
• ≤6 for 0-7 years of education.
• Note: cut-offs may be modified over time as the field evolves in this area.
• Mini-Mental State Exam score between 24 and 30 (inclusive) (Exceptions may be made for participants with less than 8 years of education at the discretion of the Project Director and/or Clinical Core)
• Clinical Dementia Rating = 0.5. Memory Box score must be at least 0.5
• General cognition and functional performance sufficiently preserved such that a diagnosis of dementia cannot be made by the site physician at the time of the screening visit.
• Stability of Permitted Medications for 4 weeks. In particular, participants may:
• Take stable doses of antidepressants lacking significant anticholinergic side effects (if they are not currently depressed and do not have a history of major depression within the past 1 year)
• Estrogen replacement therapy is permissible
• Gingko biloba is permissible, but discouraged
• Washout from psychoactive medication (e.g., excluded antidepressants, neuroleptics, chronic anxiolytics or sedative hypnotics, etc.) for at least 4 weeks prior to screening
• Cholinesterase inhibitors and memantine are allowable if stable for 12 weeks prior to screen
• Aducanumab and any other approved treatments for the neurobiology of AD if stable for 24 weeks prior to screen Inclusion Criteria for Newly Enrolled Participants, DEM Cohort
• Participant must have a subjective memory concern as reported by participant, study partner, or clinician.
• Abnormal memory function documented by scoring within the demographically- adjusted ranges on the Logical Memory II subscale (Delayed Paragraph Recall, Paragraph A only) from the Wechsler Memory Scale - Revised (the maximum score is 25):
• ≤11 for 16 or more years of education
• ≤9 for 8-15 years of education
• ≤6 for 0-7 years of education.
• Note: cut-offs may be modified over time as the field evolves in this area.
• Mini-Mental State Exam score between 20 and 28 (inclusive) (Exceptions may be made for participants with less than 8 years of education at the discretion of the Project Director and/or Clinical Core)
• Clinical Dementia Rating = 0.5 or 1.0.
• Meets the National Institute on Aging/Alzheimer's Association Diagnostic Guidelines for Dementia (2011)
• Stability of Permitted Medications for 4 weeks. In particular, participants may:
• Take stable doses of antidepressants lacking significant anticholinergic side effects (if they are not currently depressed and do not have a history of major depression within the past 1 year)
• Estrogen replacement therapy is permissible
• Gingko biloba is permissible, but discouraged
• Washout from psychoactive medication (e.g., excluded antidepressants, neuroleptics, chronic anxiolytics or sedative hypnotics, etc.) for at least 4 weeks prior to screening
• Cholinesterase inhibitors and memantine are allowable if stable for 12 weeks prior to screen
• Aducanumab and any other approved treatments for the neurobiology of AD if stable for 24 weeks prior to screen Inclusion Criteria for Newly Enrolled Participants, All Cohorts
• Geriatric Depression Scale score less than 10.
• Age between 55-90 years (inclusive).
• Study partner who has frequent contact with the participant (i.e., minimum average of 2 hours per week) and may be able to accompany the participant to clinic visits or provide information remotely (e.g. over the phone).
• Visual and auditory acuity adequate for neuropsychological testing.
• Good general health with no diseases expected to interfere with the study.
• Participant is not pregnant, lactating, or of childbearing potential (i.e., women must be two years post-menopausal or surgically sterile).
• Willing and able to participate in a longitudinal imaging study.
• Must be literate and speak English or Spanish fluently.
• Agrees to collection of blood for GWAS, APOE testing, DNA and RNA testing
• Agrees to collection of blood for biomarker testing.
• The Administrative Core, described in section 9.1.1, will collaborate with leadership from all Cores to review the blood biomarker data from the remote blood cohort and select participants to join the in-clinic cohort. See ADNI4: Remote protocol.
• Agrees to participate in the ADNI study which includes cognitive evaluation, MRI and PET scans.
• Flexibility can be made to all criteria for those with at least 8 years in a low socio-economic status (SES) neighborhood. Inclusion Criteria for Rollover Participants, All Cohorts The following additional inclusion criteria apply to all diagnostic categories for rollover participants only:
• Must have been enrolled and followed in one of the following previous ADNI studies: ADNIGO, ADNI2, ADNI3 for at least one year.
• Willing and able to continue to participant in an ongoing longitudinal study. A reduced battery of tests is allowable.
• Study partner may be available who has frequent contact with the participant (i.e., minimum average of 2 hours per week), and may be able to accompany the participant to clinic visits or provide information remotely (e.g. over the phone). Exclusion Criteria for Newly Enrolled Participants, CN Cohort:
• Any significant neurologic disease, such as Parkinson's disease, vascular cognitive impairment/dementia, Huntington's disease, normal pressure hydrocephalus, brain tumor, progressive supranuclear palsy, seizure disorder, subdural hematoma, multiple sclerosis, or history of significant head trauma followed by persistent neurologic defaults or known structural brain abnormalities Exclusion Criteria for Newly Enrolled Participants, MCI and DEM Cohorts:
• Any significant neurologic disease other than suspected Alzheimer's disease, such as Parkinson's disease (Parkinsonian symptoms complicating MCI/AD are acceptable), vascular cognitive impairment dementia (multiple lacunes less than or equal to 1.5 cm and/or extensive white matter changes are acceptable), Huntington's disease, normal pressure hydrocephalus, brain tumor (clinically insignificant meningioma acceptable), progressive supranuclear palsy, seizure disorder, subdural hematoma, multiple sclerosis, or history of significant head trauma followed by persistent neurologic defaults or known structural brain abnormalities. Exclusion Criteria for Newly Enrolled Participants, All Cohorts: Additional exclusion criteria apply to all diagnostic categories for newly enrolled participants:
• Screening/Baseline MRI brain scan with evidence of infection, or other clinically significant focal lesions. Participants with cortical strokes, not large enough to distort anatomy, multiple lacunar infarctions or extensive white matter disease are allowed.
• Screening/Baseline MRI brain scan with evidence of large structural abnormalities that would corrupt image analytical pipelines - e.g. large hemispheric infarcts, large areas of encephalomalacia, large arachnoid cysts
• Unable to complete MRIs for any reason (e.g. pacemaker or other implanted metal devices, severe claustrophobia, anxiety which prevents MRI scans, too large to fit, etc.).
• Current major depression, bipolar disorder as described in DMS-IV within the past 1 year. Psychotic features, agitation or behavioral problems within the last 3 months which could lead to difficulty complying with the protocol.
• Currently treated with medication for obsessive-compulsive disorder or attention deficit disorder.
• History of schizophrenia (DSM-5 criteria).
• History of alcohol or substance disorder within the past 2 years (DSM-5 criteria).
• Any significant systemic illness or unstable medical condition which could lead to difficulty complying with the protocol.
• Clinically significant abnormalities in B12, or thyroid function tests that might interfere with the study. A low B12 is exclusionary, unless follow-up labs (homocysteine (HC) and methylmalonic acid (MMA)) indicate that it is not physiologically significant.
• Residence in skilled nursing facility
• Current use of specific psychoactive medications (e.g. certain antidepressants, neuroleptics, chronic anxiolytics or sedative hypnotics, etc.), at the discretion of the clinician.
• Current use of any other exclusionary medications.
• Investigational agents are prohibited for five half-lives or one month, whichever time period is longer, prior to entry and for the duration of the trial.
• Participation in clinical studies involving neuropsychological measures being collected more than once time per year.
• Female that is pregnant, lactating, or of childbearing potential.
• Flexibility can be made to all criteria for those with at least 8 years in a low socio-economic status (SES) neighborhood.

Interventions: RADIATION: Neuraceq, RADIATION: Amyvid, RADIATION: Tauvid, RADIATION: MK-6240, RADIATION: NAV4694, RADIATION: PI-2620

Conditions: Mild Cognitive Impairment, Alzheimer Disease, Dementia, Brain and Nervous System

Keywords: amyloid, plaques, neuroimaging, biomarkers, cognition disorder, early detection, pre-dementia, dementia, Alzheimer's disease, tau, observational

Sites: UT Southwestern

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Cognitive Outcomes of Brain Stimulation As a Later-in-Life Treatment (COBALT)

Description:

This is a pilot study being done to attempt to improve episodic memory problems in persons with mild cognitive impairment (MCI) or dementia. The pre-supplemental motor area (preSMA) and dorsal anterior cingulate cortex (dACC) have been shown to play a role in episodic memory and language retrieval. Prior studies have suggested that neurostimulation targeting this region can improve episodic memory and word recall. The purpose of this study is to examine the efficacy of high-definition transcranial direct current stimulation (HD-tDCS) to the preSMA/dACC region and its influence on word retrieval and other cognitive functions in patients with MCI or dementia. Entraining the preSMA/dACC circuit with 10 sessions of HD-tDCS will allow us to study whether neurostimulation may be an effective treatment.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, Hannah.Cabrera@UTSouthwestern.edu

Principal Investigator: Christian LoBue

Gender: ALL

Age: 55 Years and over

Phase: NA

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05564715

IRB Number: STU-2022-0799

Show full eligibility criteria

Interventions: DEVICE: NeuroElectric StarStim, DEVICE: Sham Treatment

Conditions: Mild Cognitive Impairment, Dementia, Amnestic Mild Cognitive Impairment - aMCI, Brain and Nervous System

Sites: UT Southwestern; Parkland Health & Hospital System

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ONC201 in H3 K27M-mutant Diffuse Glioma Following Radiotherapy (the ACTION Study) (ACTION)

Description:

This is a randomized, double-blind, placebo-controlled, parallel-group, international, Phase 3 study in patients with newly diagnosed H3 K27M-mutant diffuse glioma to assess whether treatment with ONC201 following frontline radiotherapy will extend overall survival and progression-free survival in this population. Eligible participants will have histologically diagnosed H3 K27M-mutant diffuse glioma and have completed standard frontline radiotherapy.

Contact(s):

Call 833-722-6237
canceranswerline@utsouthwestern.edu

Principal Investigator: Daniel Bowers

Gender: ALL

Age: Not specified

Phase: PHASE3

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05580562

IRB Number: STU-2023-0079

Show full eligibility criteria

Inclusion Criteria:

• Able to understand the study procedures and agree to participate in the study by providing written informed consent (by participant or legally authorized representative), and assent when applicable.
• Body weight ≥ 10 kg at time of randomization.
• Histologically diagnosed H3 K27M-mutant diffuse glioma (new diagnosis). Detection of a missense K27M mutation in any histone H3-encoding gene detected by testing of tumor tissue (immunohistochemistry \[IHC\] or next-generation sequencing \[NGS\] in a Clinical Laboratory Improvement Amendments \[CLIA\]-certified or equivalent laboratory). \[Site to provide (as available): ≥ 10 unstained formalin-fixed paraffin-embedded (FFPE) slides from tumor tissue.\]
• At least one, high-quality, contrast-enhanced MRI of the brain obtained prior to starting radiotherapy for submission to sponsor's imaging vendor for central read. For participants who had a surgical resection, this scan must be post-resection; for participants who did not have a resection, this scan may be pre- or post-biopsy.
• At least one, high-quality, contrast-enhanced MRI of the brain obtained 2 to 6 weeks after completion of frontline radiotherapy. If unable to obtain contrast-enhanced imaging due to lack of venous access after multiple attempts, a patient may still be eligible after collection of a nonenhanced MRI of the brain. \[Site to also provide all available MRIs completed prior to initiating treatment with study intervention.\]
• Received frontline radiotherapy
• Initiated radiotherapy within 12 weeks from the initial diagnosis of H3 K27M-mutant diffuse glioma.
• Completed radiotherapy within 2 to 6 weeks prior to randomization
• Completed standard fractionated radiotherapy (eg. 54 to 60 Gy in 28 to 33 fractions given over approximately 6 weeks or hypofractionated radiotherapy (eg. 40 Gy in 15 fractions given over approximately 3 weeks).
• Karnofsky Performance Status or Lansky Performance Status ≥ 70 at time of randomization.
• Stable or decreasing dose of corticosteroids and anti-seizure medications for 7 days prior to randomization, if applicable. Stable steroid dose is defined as ≤ 2 mg/day increase (based on dexamethasone dose or equivalent dose of an alternative steroid).

Exclusion Criteria:

• Primary spinal tumor.
• Diffuse intrinsic pontine glioma (DIPG), defined as tumors with a pontine epicenter and diffuse involvement of the pons.
• Evidence of leptomeningeal spread of disease or cerebrospinal fluid dissemination.
• Any known concurrent malignancy.
• New lesion(s) outside of the radiation field.
• Received whole-brain radiotherapy.
• Received proton therapy for glioma.
• Use of any of the following treatments within the specified time periods prior to randomization:
• ONC201 or ONC206 at any time.
• Systemic bevacizumab (includes biosimilars) at any time since the initial diagnosis of H3 K27M-mutant diffuse glioma.
• Temozolomide within past 3 weeks.
• Tumor treating fields at any time.
• DRD2 antagonist within past 2 weeks.
• Any investigational therapy within past 4 weeks.
• Strong CYP3A4 inhibitors within 3 days.
• Strong CYP3A4 inducers (includes enzyme-inducing antiepileptic drugs) within 2 weeks.
• Laboratory test results meeting any of the following parameters within 2 weeks prior to randomization:
• Absolute neutrophil count \< 1.0 × 109/L or platelets \< 75 × 109/L.
• Total bilirubin \> 1.5 × upper limit of normal (ULN) (participants with Gilbert's syndrome may be included with total bilirubin \> 1.5 × ULN if direct bilirubin is ≤ 1.5 × ULN).
• Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \> 2.5 × ULN.
• Creatinine clearance ≤ 60 mL/min as calculated by the Cockcroft Gault equation (or estimated glomerular filtration rate \< 60 mL/min/1.73 m2).
• QTc \> 480 msec (based on mean from triplicate electrocardiograms) during screening.
• Known hypersensitivity to any excipients used in the study intervention formulation.
• Pregnant, breastfeeding, or planning to become pregnant while receiving study intervention or within 3 months after the last dose. Participants of childbearing potential must have a negative serum pregnancy test within 72 hours prior to receiving the first dose of study intervention.
• Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection requiring systemic therapy or psychiatric illness/social situations that would limit compliance with study requirements.
• Any other condition (eg, medical, psychiatric, or social) that, in the opinion of the investigator, may interfere with participant safety or the ability to complete the study according to the protocol.

Interventions: DRUG: Dordaviprone (ONC201), DRUG: Dordaviprone (ONC201) + Placebo, OTHER: Placebo

Conditions: H3 K27M, Glioma, Brain and Nervous System

Keywords: H3 K27M, H3 K28M, H3 K27-altered, histone, H3F3A, HIST1H3B, HIST1H3C, H3.1, H3.3, DMG, thalamus, thalamic, midline

Sites: Children’s Health

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Molecular and Clinical Risk-Directed Therapy for Infants and Young Children With Newly Diagnosed Medulloblastoma

Description:

This is a multi-center, multinational phase 2 trial that aims to explore the use of molecular and clinical risk-directed therapy in treatment of children 0-4.99 years of age with newly diagnosed medulloblastoma.

Contact(s):

Call 833-722-6237
canceranswerline@utsouthwestern.edu

Principal Investigator: Daniel Bowers

Gender: ALL

Age: to 59 Months old

Phase: PHASE2

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05535166

IRB Number: STU-2023-0119

Show full eligibility criteria

Inclusion Criteria - Screening Phase (All Patients) * Participants with presumptive/suspected newly diagnosed medulloblastoma. * Participant meets one of the following criteria at the time of screening: * Age \< 36 months OR Age ≥ 36 months and \< 60 months with presumptive/suspected non-metastatic disease * Participant must have adequate tumor tissue from primary tumor for central review of pathology and molecular classification by methylation and IHC * Participant must be able to begin treatment as outlined in the protocol within 36 days of definitive surgery (day of surgery is Day 0). In case a second surgery is clinically indicated to remove the residual tumor prior to starting treatment, the second surgery will be considered as the definitive surgery (Day 0). * Parent or legal guardian can understand and is willing to sign a written informed consent document according to institutional guidelines. Exclusion Criteria - Screening Phase * Participants with other clinically significant medical disorders (i.e., serious infections or significant cardiac, pulmonary, hepatic, psychiatric, or other organ dysfunction) that could compromise their ability to tolerate protocol therapy or would interfere with the study procedure. Inclusion Criteria - Study Enrollment (All Patients) * Participant must be \< 60 months of age at time of enrollment. * Note: Each treatment stratum has additional specific age requirements * Participant must have confirmation of newly diagnosed medulloblastoma per Central Review: * Central review includes histopathology, IHC and St. Jude Clinical Genomic Methylation Profiling conducted on MLPNet. If tissue or the extracted DNA does not meet quality control criteria for methylation analysis or if methylation classifier is unable assign molecular group/subgroup within the assigned classifier (MLPNet) parameters, then IHC will be used to define molecular group of these cases. IHC cannot be used to determine molecular subgroup. Therefore, IHC defined SHH patients will be enrolled on Stratum S-1 under "SHH-NOS", and all NWNS and indeterminate molecular group will be enrolled on stratum N. * Note: Diagnosis of medulloblastoma, as well as group and subgroup assignment, will be done by central pathology review at St. Jude only. No outside testing is allowed for trial enrollment. * Participant must have disease staged by MRI of the brain and spine and by cytologic examination of CSF\* and be placed into the following categories: * M0: no evidence of metastatic disease. * must include a negative CSF cytology result * M1: Tumor cells found in the CSF but no other evidence of metastasis * M2: Intracranial tumor beyond the primary tumor site * M3: Metastatic disease in the spine * M4: Extraneural metastatic disease * \*All participants are to undergo CSF cytologic examination regardless of presence or absence of gross metastatic disease unless procedure is medically contraindicated. CSF is to be obtained by lumbar puncture (LP) performed at least 10 days after surgery. If LP is medically contraindicated, ventricular CSF from a shunt or Ommaya reservoir may be used for staging but this is not the preferred option due to lower sensitivity. If LP is medically contraindicated and the patient doesn't have a shunt or reservoir for CSF sampling, the treating physician should reach out to PI or Co-PI regarding decision on enrollment to SJiMB21. The decision to enroll without CSF cytology will be made on case-by-case basis. * Note: Participants who have M2 disease and positive CSF will be assigned to M3. * Note: Participants will be assigned to the highest stage number for which they meet eligibility. * Note: Treatment stratums may have additional stage requirements. * Patient must have received no previous radiotherapy, chemotherapy, or other brain tumor-directed therapy other than corticosteroid therapy and surgery. * Participant must have a Lansky performance score of \> 30 (except for patients with posterior fossa syndrome. * Participant must have adequate organ function prior to study entry, as defined by: * Absolute neutrophil counts (ANC) \>750/mm\^3 * Platelet count ≥ 50,000/mm\^3 without support of a platelet transfusion within 7 days * Hemoglobin ≥8.0 g/dL (with or without support of a blood transfusion). * Normal liver function as defined by Alanine aminotransferase (ALT) concentration ≤ 3 x 45 U/L and total bilirubin ≤ 3 x 1.0. * Adequate renal function as defined by a serum creatinine concentration: * Age - 0 to \<1year; Maximum Serum Creatinine (mg/dl) - Male 0.5; Female 0.5 * Age - 1 to \< 2years; Maximum Serum Creatinine (mg/dl) - Male 0.6; Female 0.6 * Age - 1 to \< 2yearsr; Maximum Serum Creatinine (mg/dl) - Male 0.8; Female 0.8 * Participant's parent or legal guardian has the ability to understand and the willingness to sign a written informed consent document according to institutional guidelines. Inclusion Criteria - Stratum S-2 * Participant must have confirmed diagnosis of the following medulloblastoma molecular group and subgroup per Central Review. * Medulloblastoma SHH-2 * Participant must meet one of the following criteria at time of enrollment: * Age \<36 months OR Age ≥ 36 months and \< 60 months with non-metastatic disease (M0) Inclusion Criteria - Stratum S-1 * Participant must have confirmed diagnosis of one of the following medulloblastoma molecular subgroups per Central Review. * Medulloblastoma SHH-1 * Medulloblastoma SHH-3 * Medulloblastoma SHH-4 * Medulloblastoma SHH-NOS * Includes medulloblastoma cases that could not be assigned to a molecular subgroup using the DNA methylation classifier, but which are in the SHH group and/or cases defined as SHH by IHC. * Participant must be \< 36 months of age at time of enrollment * Note: Patients who are \< 36 months of age, regardless of metastatic status (M0/M+), are eligible for enrollment on stratum S-1. Inclusion Criteria - Stratum N * Participant must have confirmed diagnosis of one of the following medulloblastoma molecular subgroups per Central Review. * Medulloblastoma G3 * Medulloblastoma G4 * Medulloblastoma - Not classified into SHH (i.e., NWNS or indeterminate) * Includes medulloblastoma cases that could not be assigned to a molecular group using the DNA methylation classifier but which are in the NWNS class and/or defined as NWNS by IHC. * Participant must be \<36 months of age at time of enrollment * All NWNS patients (M+ and M0) are eligible for enrollment in stratum N Exclusion Criteria - All Patients * CNS embryonal tumor other than medulloblastoma, for example, patients with diagnosis of Atypical Teratoid/Rhabdoid Tumor (ATRT), PNET, Pineoblastoma, Ependymoma, and ETMR are excluded. * Participant with prior treatment for medulloblastoma, including: * Radiotherapy * Chemotherapy * Cancer directed immunotherapy * Targeted agents * NOTE: Corticosteroid therapy is acceptable; prior treatment with chemotherapy, immunotherapy or targeted agents for non-cancer directed indications are acceptable as long as these have been stopped at least 14 days prior to start of therapy or 2 half-lives from last dose. (i.e., methotrexate for juvenile rheumatoid arthritis, JAK inhibitor therapy for eczema, etc.) * Participant who is actively receiving any other investigational agents. * Participant with other clinically significant medical disorders (i.e., serious infections or significant cardiac, pulmonary, hepatic, psychiatric, or other organ dysfunction) that could compromise their ability to tolerate protocol therapy or would interfere with the study procedures or results.

Interventions: PROCEDURE: Surgical resection, PROCEDURE: Ommaya/VPS, DRUG: Methotrexate, DRUG: Cisplatin, DRUG: Vincristine, DRUG: Cyclophosphamide, DRUG: Carboplatin, DRUG: Topotecan, DRUG: Etoposide, DRUG: Pegfilgrastim, DRUG: Filgrastim, RADIATION: Irradiation, OTHER: Educational and Media Intervention, OTHER: SOC, Educational and Media Intervention

Conditions: Medulloblastoma, Brain and Nervous System

Keywords: SJiMB21, Brain Cancer, Brain Tumors in Children, Medulloblastoma Sonic Hedgehog subgroup 1, Medulloblastoma Sonic Hedgehog subgroup 2, Medulloblastoma Sonic Hedgehog subgroup 3, Medulloblastoma Sonic Hedgehog subgroup 4, Medulloblastoma Sonic Hedgehog-not otherwise specified, Medulloblastoma G3, Medulloblastoma G4, Medulloblastoma indeterminate, MLPNet, Neural Net Classification Pipeline, Non-WNT non-SHH medulloblastoma, Posterior fossa syndrome, St. Jude Brain Tumor Studies, Treatment for Brain Tumors in Infants and Young Children, Untreated Childhood Medulloblastoma

Sites: Children’s Health

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A Study to Evaluate the Safety and Efficacy of CT1812 in Early Alzheimer's Disease

Description:

This is a multicenter randomized, double-blind, placebo-controlled Phase 2 study designed to evaluate the efficacy, safety, and tolerability of two doses of CT1812 compared to placebo in participants diagnosed with early Alzheimer's disease.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, Matthew.Jones@UTSouthwestern.edu

Principal Investigator: Brendan Kelley

Gender: ALL

Age: 50 Years to 85 Years old

Phase: PHASE2

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05531656

IRB Number: STU-2024-0467

Show full eligibility criteria

Interventions: DRUG: CT1812, DRUG: Placebo

Conditions: Early Alzheimer's Disease, Brain and Nervous System

Keywords: Alzheimer's Disease

Sites: UT Southwestern

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Subclinical Transthyretin Cardiac Amyloidosis in V122I TTR Carriers

Description:

Approximately 1.5 million of the 44 million Blacks in the United States are carriers of the valine-to-isoleucine substitution at position 122 (V122I) in the transthyretin (TTR) protein. Virtually exclusive to Blacks, this is the most common cause of hereditary cardiac amyloidosis (hATTR-CA) worldwide. hATTR-CA leads to worsening heart failure (HF) and premature death. Fortunately, new therapies that stabilize TTR improve morbidity and mortality in hATTR-CA, especially when prescribed early in the disease. However, hATTR-CA is often diagnosed at an advanced stage and conventional diagnostic tools lack diagnostic specificity to detect early disease. The overall objectives of this study are to determine the presence of subclinical hATTR-CA and to identify biomarkers that indicate amyloid progression in V122I TTR carriers. The central hypothesis of this proposal is that hATTR-CA has a long latency period that will be detected through subclinical amyloidosis imaging and biomarker phenotyping. The central hypothesis will be tested by pursuing 2 specific aims: Aim 1) determine the association of V122I TTR carrier status with CMRI evidence of amyloid infiltration; Sub-aim 1) determine the association of V122I TTR carrier status with cardiac reserve; Aim 2) determine the association between amyloid-specific biomarkers and V122I TTR carrier status; and Sub-aim 2) determine the association of amyloid-specific biomarkers with imaging-based parameters and evaluate their diagnostic utility for identifying subclinical hATTR-CA. In Aim 1, CMRI will be used to compare metrics associated with cardiac amyloid infiltration between a cohort of V122I TTR carriers without HF formed by cascade genetic testing and age-, sex-, and race-matched non-carrier controls. For Sub-Aim 1, a sub-sample of carriers and non-carrier controls enrolled in Aim 1 will undergo novel exercise CMRI to measure and compare cardiac systolic and diastolic reserve. Aim 2 involves measuring and comparing amyloid-specific biomarkers in V122I TTR carriers without HF with samples matched non-carriers (both from Aim 1) and individuals with symptomatic V122I hATTR-CA from our clinical sites. These biomarkers detect and quantify different processes of TTR amyloidogenesis and include circulating TTR, retinol binding protein 4, TTR kinetic stability, and misfolded TTR oligomers. Sub-aim 2 will establish the role of these biomarkers to detect imaging evidence of subclinical hATTR-CA disease.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, Amy.Browning@UTSouthwestern.edu

Principal Investigator: Justin Grodin

Gender: ALL

Age: 30 Years to 80 Years old

Phase:

Healthy Volunteers:

System ID: NCT05489549

IRB Number: STU-2022-0404

Show full eligibility criteria

(V122I TTR carriers (or matched non-carriers))

Inclusion Criteria:

* Men and women ages 30-80 who are V122I TTR carriers (or matched non-carriers) without history of HF (this will be assessed by study personnel) and defined as: a) No history of hospitalization within the previous 12 months for management of HF; b) Without an elevated B-type natriuretic peptide level ≥100 pg/mL or NT-proBNP ≥360 pg/mL within the previous 12 months; or c) No clinical diagnosis of HF from a treating clinician * Signed informed consent

Exclusion Criteria:

* A self-reported history or clinical history of HF * Other known causes of cardiomyopathy * History of light-chain cardiac amyloidosis * Prior type 1 myocardial infarction (non-ST segment elevation myocardial Infarction {NSTEMI} or ST-elevation myocardial infarction {STEMI}) * Cardiac transplantation * Body weight \>250 lbs * Estimated glomerular filtration rate ≤30 mL/min/1.73 m2 * Inability to safely undergo CMRI (For participants with symptomatic V122I hATTR-CA, we will enroll probands with HF from Aim 1 or patients with suspected symptomatic V122I hATTR-CA from the three study sites.)

Inclusion Criteria:

* Men and women ages 30-80 who have symptomatic V122I hATTR-CA as determined by a history of HF (this will be assessed by study personnel) and defined as: a) History of hospitalization within the previous 12 months for management of HF; b) An elevated B-type natriuretic peptide level ≥100 pg/mL or NT-proBNP ≥360 pg/mL within the previous 12 months; or c) A clinical diagnosis of HF from a treating clinician. * Have an established or suspected diagnosis of hATTR-CA based on either a) Biopsy confirmed by Congo red (or equivalent) staining with tissue typing with immunohistochemistry or mass spectrometric analysis or immunoelectron microscopy, OR b) positive technetium-99m (99mTc)-pyrophosphate or -bisphosphonate scan, combined with accepted laboratory criteria without abnormal M-protein. * TTR gene sequencing that is pending or that is confirming the V122I variant * Signed informed consent

Exclusion Criteria:

* Other known causes of cardiomyopathy * History of light-chain cardiac amyloidosis * Cardiac transplantation * Liver transplantation * Previous treatment with a TTR stabilizer (tafamidis, acoramidis) within the prior 14 days or TTR any silencer (inotersen, patisiran, eplontersen) * Estimated glomerular filtration rate ≤30 mL/min/1.73 m2

Conditions: Amyloidosis, Hereditary, Amyloidosis Cardiac, Amyloidosis, Familial, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Transthyretin Gene Mutation, Cardiovascular

Keywords: Amyloidosis, Transthyretin Amyloidosis, V122I TTR, p.Val142Ile TTR, Cardiac magnetic resonance imaging

Sites: UT Southwestern

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Pathways Relating Amnestic MCI to a Mild Traumatic Brain Injury History (PATH)

Description:

This study will probe if the biological changes in amnestic mild cognitive impairment (aMCI) are related to a history of mild traumatic brain injury (mTBI) using high definition transcranial direct current stimulation (HD-tDCS) and blood-derived biomarker tools. Participants who Do as well as those who Do Not have a history of mTBI will be enrolled in the study.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, Hannah.Cabrera@UTSouthwestern.edu

Principal Investigator: Christian LoBue

Gender: ALL

Age: 55 Years and over

Phase: PHASE2

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05446584

IRB Number: STU-2022-0591

Show full eligibility criteria

Interventions: DEVICE: High Definition Transcranial Direct Current Stimulation

Conditions: Mild Cognitive Impairment, Amnestic Mild Cognitive Disorder, Amnestic Mild Cognitive Impairment, Mild Traumatic Brain Injury, Concussion, Brain, Brain and Nervous System

Keywords: MCI, TBI, memory, biomarker, Alzheimer

Sites: UT Southwestern; Parkland Health & Hospital System

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Blood-Brain Barrier Disruption (BBBD) for Liquid Biopsy in Subjects With GlioBlastoma Brain Tumors

Description:

The purpose of this study is to evaluate the safety and efficacy of targeted blood brain barrier disruption with Exablate Model 4000 Type 2.0/2.1 for liquid biopsy in subjects with suspected Glioblastoma brain tumors

Contact(s):

Call 833-722-6237
canceranswerline@utsouthwestern.edu

Principal Investigator: Bhavya Shah

Gender: All

Age: 18 Years to 80 Years old

Phase: N/A

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05383872

IRB Number: STU-2022-0890

Show full eligibility criteria

Inclusion Criteria:

• Male or Female between >18-80 years of age who are able and willing to give informed consent
• Subjects with stereotactically-targetable suspected glioblastoma tumor on pre-operative brain imaging scans
• Subjects that are scheduled, or will be scheduled within 4 weeks, for surgical resection or biopsy per standard clinical tumor care
• Karnofsky Performance Score >70
• Able to communicate sensations during the Exablate BBBD procedure

Exclusion Criteria:

• Tumor originating from the deep midline, thalamus, midbrain, cerebellum or brainstem.
• Multifocal tumors
• Tumor morphology or other imaging findings that precludes the ability to sonicate the planned tumor volume (including significant tumor volume outside the treatment envelope or tumor volume that exceeds the maximum sonication volume allowed, i.e. currently 110 ccs at the treatment volume level). Concern for adequate tumor coverage by sonication based on tumor morphology should be discussed with the Sponsor.
• MRI or clinical findings of:
• Active or chronic infection(s) or inflammatory processes
• Acute or chronic hemorrhages, specifically any lobar microbleeds, and no siderosis, amyloid angiopathy, or macro-hemorrhages
• Intracranial thrombosis, vascular malformation, cerebral aneurysm or vasculitis
• MR non-compatible metallic implants in the skull or the brain or the presence of unknown MR unsafe devices
• Significant cardiac disease or unstable hemodynamic status
• Documented myocardial infarction within six months of enrollment
• Unstable angina on medication
• Unstable or worsening congestive heart failure
• Left ventricular ejection fraction below the lower limit of normal
• History of a hemodynamically unstable cardiac arrhythmia
• Cardiac pacemaker
• History of hypersensitivity to Perflutren lipid microsphere or its components, e.g., polyethylene glycol
• Uncontrolled hypertension (systolic > 180 and diastolic BP > 120 on medication)
• Unable to discontinue use of anti-coagulant/antiplatelet therapy as per local standard.
• History of a liver disease, bleeding disorder, coagulopathy or a history of spontaneous hemorrhage or evidence of increased risk of bleeding
• Abnormal coagulation profile (Platelets < 80,000), PT (>14) or PTT (>36), and INR >
• 3
• Known cerebral or systemic vasculopathy
• Significant depression and at potential risk of suicide
• Known sensitivity/allergy to gadolinium or DEFINITY,
• Active seizures despite medication treatment (defined as >1 seizure per week) which could be worsened by disruption of the blood brain barrier
• Active drug or alcohol disorder which have a higher risk for seizures, infection and/or poor executive functioning
• Positive HIV status, which can lead to increased entry of HIV into the brain parenchyma leading to HIV encephalitis
• Potential blood-borne infections which can lead to increased entry to brain parenchyma leading to meningitis or brain abscess
• Any contraindications to MRI scanning, including:
• Large subjects not fitting comfortably into the scanner
• Difficulty lying supine and still for up to 3 hours in the MRI unit or claustrophobia
• Impaired renal function with estimated glomerular filtration rate <30 mL/min/1.73m2
• Severe Respiratory Illness: chronic pulmonary disorders e.g. severe emphysema, pulmonary vasculitis, or other causes of reduced pulmonary vascular cross-sectional area, subjects with a history of severe drug allergies, asthma or hay fever, and multiple allergies where the benefit/risk of administering Definity® is considered unfavorable by the study physicians in relation to the product labeling for Definity
• Currently in a clinical trial involving an investigational product or non-approved use of a drug or device
• Pregnancy or Lactation

Interventions: Device: Focused Ultrasound (Exablate Model 4000)

Conditions: Glioblastoma, Glioma, Liquid Biopsy, Brain and Nervous System

Sites: UT Southwestern

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Pediatric Influence of Cooling Duration on Efficacy in Cardiac Arrest Patients (P-ICECAP) (ICECAP)

Description:

This is a multicenter trial to establish the efficacy of cooling and the optimal duration of induced hypothermia for neuroprotection in pediatric comatose survivors of cardiac arrest. The study team hypothesizes that longer durations of cooling may improve either the proportion of children that attain a good neurobehavioral recovery or may result in better recovery among the proportion already categorized as having a good outcome.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, Kirstie.LeDoux@UTSouthwestern.edu

Principal Investigator: Joshua Wolovits

Gender: All

Age: 2 Days to 17 Years old

Phase: N/A

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05376267

IRB Number: STU-2022-0800

Show full eligibility criteria

Interventions: Device: Therapeutic Hypothermia

Conditions: Cardiac Arrest, Out-Of-Hospital, Hypothermia, Induced, Hypoxia-Ischemia, Brain

Keywords: Bayesian Adaptive Clinical Trial, Hypothermia, therapeutic, Coma, Pediatric

Sites: Children’s Health

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Validation of Early Prognostic Data for Recovery Outcome After Stroke for Future, Higher Yield Trials (VERIFY)

Description:

VERIFY will validate biomarkers of upper extremity (UE) motor outcome in the acute ischemic stroke window for immediate use in clinical trials, and explore these biomarkers in acute intracerebral hemorrhage. VERIFY will create the first multicenter, large-scale, prospective dataset of clinical, transmagnetic stimulation (TMS), and MRI measures in the acute stroke time window.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, HEATHER.PAUP@UTSOUTHWESTERN.EDU

Principal Investigator: Benjamin Nguyen

Gender: All

Age: 18 Years and over

Phase:

Healthy Volunteers:

This study is also accepting healthy volunteers

System ID: NCT05338697

IRB Number: STU-2021-1134

Show full eligibility criteria

Inclusion Criteria:

• Age 18 years or older
• Unilateral stroke due to ischemia or intracerebral hemorrhage
• Motor deficits in the acutely affected UE, defined as a Shoulder Abduction and Finger Extension (SAFE) score ≤ 8 out of 10 points (i.e., excluding full or nearly full motor strength in both shoulder abduction and finger extension) within 48 to 96 hours of stroke onset (or time last known well).
• Provision of signed and dated informed consent form within 48 to 96 hours of stroke onset (or time last known well).
• Stated willingness to comply with all study procedures and availability for the duration of the study
• Fluent in English or Spanish

Exclusion Criteria:

• UE injury or conditions on paretic side that limited use prior to the stroke.
• Legally blind.
• Dense sensory loss indicated by a score of 2 on NIHSS sensory item
• Unable to abduct the shoulder or extend the fingers of the non-paretic arm/hand/wrist on verbal command
• Isolated cerebellar stroke
• Bilateral hemisphere acute strokes
• Co-enrollment in a trial of an intervention targeting the incident stroke (acute treatment or rehabilitation/recovery intervention) after baseline assessments for VERIFY are initiated
• Known or expected inability to maintain follow-up with study procedures through 90 days
• Cognitive or communication impairment precluding informed consent by the participant.
• Major medical, neurological, or psychiatric condition that would substantially affect functional status
• Non-cerebrovascular diagnosis associated with unlikely survival at 90 days
• Pregnancy
• Contraindication to noncontrast MRI (i.e., certain metallic implants, metallic foreign bodies or severe claustrophobia)
• Contraindication to TMS (i.e., cardiac pacemaker or other electronic devices in the body at or above the level of the seventh cervical vertebra, such as cochlear implant, cortical stimulator, deep brain stimulator, vagus nerve stimulator, cervical spine epidural stimulator, or ventriculoperitoneal shunt; Skull defect related to current stroke; Seizure after onset of current stroke; Seizure within the last 12 months while taking anti-epileptic medications; Previous serious adverse reaction to TMS)
• Unable to perform behavioral assessments within 48-120 hours of symptom onset
• Unable to receive TMS or get MRI within 72-168 hours of symptom onset
• Anticipated inability to perform study procedures within 168 hours of symptom onset.

Interventions: Diagnostic Test: Transcranial Magnetic Stimulation (TMS)

Conditions: Stroke, Stroke, Acute, Stroke, Ischemic, Stroke Hemorrhagic, Brain and Nervous System, Other

Sites: UT Southwestern

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A Study of Treatment for Medulloblastoma Using Sodium Thiosulfate to Reduce Hearing Loss

Description:

This phase III trial tests two hypotheses in patients with low-risk and average-risk medulloblastoma. Medulloblastoma is a type of cancer that occurs in the back of the brain. The term, risk, refers to the chance of the cancer coming back after treatment. Subjects with low-risk medulloblastoma typically have a lower chance of the cancer coming back than subjects with average-risk medulloblastoma. Although treatment for newly diagnosed average-risk and low-risk medulloblastoma is generally effective at treating the cancer, there are still concerns about the side effects of such treatment. Side effects or unintended health conditions that arise due to treatment include learning difficulties, hearing loss or other issues in performing daily activities. Standard therapy for newly diagnosed average-risk or low-risk medulloblastoma includes surgery, radiation therapy, and chemotherapy (including cisplatin). Cisplatin may cause hearing loss as a side effect. In the average-risk medulloblastoma patients, this trial tests whether the addition of sodium thiosulfate (STS) to standard of care chemotherapy and radiation therapy reduces hearing loss. Previous studies with STS have shown that it may help reduce or prevent hearing loss caused by cisplatin. In the low-risk medulloblastoma patients, the study tests whether a less intense therapy (reduced radiation) can provide the same benefits as the more intense therapy. The less intense therapy may cause fewer side effects. Radiation therapy uses high energy x-rays to kill tumor cells and shrink tumors. Cisplatin is in a class of medications known as platinum-containing compounds. It works by killing, stopping or slowing the growth of cancer cells. The overall goals of this study are to see if giving STS along with standard treatment (radiation therapy and chemotherapy) will reduce hearing loss in medulloblastoma patients and to compare the overall outcome of patients with medulloblastoma treated with STS to patients treated without STS on a previous study in order to make sure that survival and recurrence of tumor is not worsened.

Contact(s):

Call 833-722-6237
canceranswerline@utsouthwestern.edu

Principal Investigator: Daniel Bowers

Gender: ALL

Age: 4 Years to 21 Years old

Phase: PHASE3

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05382338

IRB Number: STU-2023-0231

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Inclusion Criteria:

* PRE-ENROLLMENT: Patients must be ≥ 4 years and ≤ 21 years of age at the time of enrollment * PRE-ENROLLMENT: Patient is suspected to have newly-diagnosed medulloblastoma by institutional diagnosis * Please note: Patients with a pending result of CSF cytology tests are eligible for NCI-2014-02057 (APEC14B1-Central Nervous System \[CNS\]) and CNS/Medulloblastoma Pre Enrollment Eligibility Screening * PRE-ENROLLMENT: The patient and/or their parents or legal guardians must have signed informed consent for APEC14B1 Part A - Eligibility Screening and consent for the Molecular Characterization Initiative (MCI) * PRE-ENROLLMENT: The required specimens are projected to be submitted under APEC14B1-CNS as soon as possible, preferably within 5 days of definitive surgery * PRE-ENROLLMENT: All patients must have rapid central pathology review under APEC14B1-CNS prior to study enrollment on ACNS2031 step 1 in order to avoid discordant diagnoses and to verify diagnosis criterion for treatment on ACNS2031. * Note: Patients with a pending result of CSF cytology tests are eligible for the rapid central pathology screening review. Confirmation of CSF negativity is needed for enrollment on the ACNS2031 protocol * PRE-ENROLLMENT: All patients must have rapid central molecular screening review under APEC14B1-CNS prior to study enrollment on ACNS2031 step 1, in order to avoid discordant diagnoses and to verify diagnosis criterion for treatment on ACNS2031 * PRE-ENROLLMENT: All patients who have histopathology confirmed must have rapid central imaging screening review under APEC14B1 prior to study enrollment on ACNS2031 step 1 * Note: Patients must not have metastatic disease on cranial or spinal MRI. Patients with \> 1.5 cm\^2 residual tumor after initial surgical resection may undergo a second surgical resection prior to subsequent therapy to render them eligible for this study. The day of the second resection to remove residual tumor will be regarded as the day of definitive surgery (Day 0) and must be within a month (31 days) of the initial resection * PRE-ENROLLMENT: All patients who have histopathology confirmed must have rapid central audiology review under APEC14B1-CNS prior to study enrollment on ACNS2031 step 1 * Patients must be \>= 4 years and =\< 21 years of age at the time of enrollment * Patients must be newly diagnosed and have eligibility confirmed by rapid central pathology and molecular screening reviews performed on APEC14B1 and via the Molecular Characterization Initiative * Average-risk cohort * Clinico-pathologic criteria: * M0 disease * No diffuse anaplastic histology AND * Molecular criteria: * SHH, p53wt, GLI2 normal, MYCN normal, no chromosome 14q loss * Group 3, MYC normal, no isochromosome 17q * Group 4, no chromosome 11 loss * Low-risk features cohort * Clinico-pathologic criteria: * M0 disease * No diffuse anaplastic histology AND * Molecular criteria: * Group 4, chromosome 11 loss * Patients must have negative lumbar CSF cytology * Note: CSF cytology for staging should be performed no sooner than 14 days post operatively to avoid false positive CSF. Ideally, CSF should be obtained between day 14 and day 21 to allow for final staging status before enrollment onto the study. Patients with positive CSF cytology obtained 0 to 14 days after surgery should have cytology repeated to determine eligibility and final CSF status. Patients with negative CSF cytology from lumbar puncture obtained 0 to 14 days after surgery do not need cytology repeated. Patients with negative CSF cytology from lumbar puncture obtained prior to surgery do not need cytology repeated post-operatively * Patients must have eligibility confirmed by Rapid Central Imaging Review performed on APEC14B1. Patients must have =\< 1.5 cm\^2 cross-sectional area of residual tumor. Whole brain MRI with and without gadolinium and spine MRI with gadolinium must be performed * Patients must weigh \> 10 kg * Patients must be enrolled, and protocol therapy must be projected to begin, no later than 31 days after definitive diagnostic surgery (day 0) * Peripheral absolute neutrophil count (ANC) \>= 1000/uL (within 7 days prior to enrollment) * Platelet count \>= 100,000/uL (transfusion independent) (within 7 days prior to enrollment) * Hemoglobin \>= 8.0 g/dL (may receive red blood cell count \[RBC\] transfusions) (within 7 days prior to enrollment) * A serum creatinine (within 7 days prior to enrollment) based on age/sex as follows: * 4 to \< 6 years (age); 0.8 mg/dL (male) 0.8 mg/dL (female) * 6 to \< 10 years (age); 1 mg/dL (male) 1 mg/dL (female) * 10 to \< 13 years (age); 1.2 mg/dL (male) 1.2 mg/dL (female) * 13 to \< 16 years (age); 1.5 mg/dL (male) 1.4 mg/dL (female) * \>= 16 years (age); 1.7 mg/dL (male) 1.4 mg/dL (female) OR a 24 hour urine Creatinine clearance \>= 70 mL/min/1.73 m\^2 (within 7 days prior to enrollment) OR a glomerular filtration rate (GFR) \>= 70 mL/min/1.73 m\^2 (within 7 days prior to enrollment). GFR must be performed using direct measurement with a nuclear blood sampling method OR direct small molecule clearance method (iothalamate or other molecule per institutional standard) * Note: Estimated GFR (eGFR) from serum creatinine, cystatin C or other estimates are not acceptable for determining eligibility * Total bilirubin =\< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to enrollment) * Serum glutamic pyruvic transaminase (SGPT) (alanine aminotransferase \[ALT\]) =\< 135 U/L (within 7 days prior to enrollment) * Note: For the purpose of this study, the ULN for SGPT (ALT) has been set to the value of 45 U/L * Central nervous system function defined as: * Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled * Patients must not be in status epilepticus, a coma or assisted ventilation at the time of study enrollment * Auditory function defined as: * Patients must have normal hearing (defined as International Society of Pediatric Oncology \[SIOP\] grade 0) in at least one ear confirmed by rapid central audiology review performed on APEC14B1 prior to enrollment * All patients and/or their parents or legal guardians must sign a written informed consent * All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met

Exclusion Criteria:

* Patients with metastatic disease by either MRI evaluation or lumbar CSF cytology are not eligible. Patients who are unable to undergo a lumbar puncture for assessment of CSF cytology are ineligible * Patients must not have received any prior radiation therapy or chemotherapy (tumor-directed therapy) other than surgical intervention and/or corticosteroids * Patients must not have any known hypersensitivity to STS, sulfates/sulfites, or other thiol agents (e.g., amifostine, n-acetylcysteine, MESNA, and captopril) * Pregnancy and Breastfeeding: * Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential * Lactating females who plan to breastfeed their infants * Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation

Interventions: PROCEDURE: Audiometric Test, PROCEDURE: Auditory Brainstem Response, PROCEDURE: Biospecimen Collection, DRUG: Cisplatin, DRUG: Cyclophosphamide, DRUG: Lomustine, PROCEDURE: Magnetic Resonance Imaging, OTHER: Quality-of-Life Assessment, RADIATION: Radiation Therapy, DRUG: Sodium Thiosulfate, OTHER: Survey Administration, DRUG: Vincristine

Conditions: Childhood Medulloblastoma, Brain and Nervous System

Sites: Children’s Health

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Impact of Intensive Treatment of SBP on Brain Perfusion, Amyloid, and Tau (IPAT Study) (IPAT)

Description:

The purpose of this study is to determine if intensive lowering of systolic blood pressure (SBP), using FDA approved medications (antihypertensive), reduces Alzheimer's Disease pathology (i.e., excessive brain amyloid and tau protein deposition) in older adults at high risk for memory decline or dementia.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, TristynHall@texashealth.org

Principal Investigator: Rong Zhang

Gender: ALL

Age: 60 Years to 85 Years old

Phase: PHASE2

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05331144

IRB Number: STU-2021-1210

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Inclusion Criteria:

* Age 60-85, all races/ethnicities, and both sexes are eligible; * Mini-Mental State Exam (MMSE) ≥ 26 to exclude gross dementia; based on clinical judgment, may be rescreened in ≥ 7 days; * Individuals with SBP ≥ 130 and SBP ≤ 180 if on 0 or 1 antihypertensive medications; ≥130 and ≤170 on up to 2 medications; ≥130 and ≤160 on up to 3 medications; ≥130 and ≤150 on up to 4 medications. Those on antihypertensives are eligible. If an individual, not treated for hypertension (HTN), has a SBP ≥ 125 mmHg, consider rescreening after 24 hours; * Willingness to be randomized into the treatment groups and ability to return to clinic for follow-up visits over 24 months; * Fluency in English or Spanish or both, adequate visual and auditory acuity to allow neuropsychological testing; * Participants must have a regular healthcare provider.

Exclusion Criteria:

* Clinically documented history of stroke, focal neurological signs or other major cerebrovascular diseases based on clinical judgment or MRI/CT scans such as evidence of infection, infarction, or other brain lesions; * Diagnosis of AD or other type of dementia, or significant neurologic diseases such as Parkinson's disease, seizure disorder, multiple sclerosis, history of severe head trauma or normal pressure hydrocephalus; * Evidence of severe major depression (GDS ≥ 12, may be rescreened after 12 weeks or longer if evidence of reactive depression or temporary mood disturbances) or clinically significant psychopathology, (e.g., psychosis and schizophrenia); if hospitalized in past year, can be rescreened in 6 months; or presence of a major psychiatric disorder that in the investigator's opinion, could interfere with adherence to research assessments or procedures. * Unstable heart disease based on clinical judgment (e.g., heart attack/cardiac arrest, cardiac bypass procedures within previous 6 months and congestive heart failure), or other severe medical conditions; * History of atrial fibrillation and evidence on ECG with any of the following: active symptoms of persistent palpitation, dizziness, history of syncope, chest pain, dyspnea, orthopnea, shortness of breath at rest, or paroxysmal nocturnal dyspnea within the past 6 months; resting heart rate of \< 30 or \> 110 bpm; taking class I or III antiarrhythmic drugs including flecainide, propafenone, dronedarone, sotalol, dofetilide, and amiodarone; or clinical concerns for safely participating in lowering blood pressure. * Systolic BP equal or greater than 180 mmHg and/or diastolic BP equal or greater than 110 mmHg, may be rescreened in 1 week. * Orthostatic hypotension, defined as the third standing SBP \< 100mmHg, may be rescreened after 2 weeks; * History of significant autoimmune disorders such as systemic lupus erythematosus, rheumatoid arthritis or polymyalgia rheumatica; * Significant history of alcoholism or drug abuse within the last five years; * Uncontrolled diabetes mellitus, defined as hemoglobin A1C \> 7.5%, or requiring insulin treatment; * Regularly smoking cigarettes within the past year; * Pacemaker or other medical device of metal that precludes performing MRI; * Women with a potential for pregnancy, lactation/childbearing (2 year post-menopausal or surgically sterile to be considered not childbearing potential); * Participant enrolled in another investigational drug or device study, either currently or within the past 2 months; * Severe obesity with BMI \> 40 ; clinical judgment should be applied in all cases to assess patient safety and anticipated compliance; * Allergy to angiotensin receptor blockers (ARBs), i.e., drugs that have a suffix "-sartan"; allergy to amlodipine; * Abnormal screening laboratory tests (e.g., liver ALT and AST \> 3 x ULN, GFR \< 30 or Hct \< 28%); may be rescreened after 2 weeks or longer; * A medical condition likely to limit survival to less than 3 years; * Participant has any condition(s) judged by the study investigator to be medically inappropriate, risky or likely to cause poor study compliance. For example:
• Plans to move outside the clinic catchment area in the next 2 years;
• Significant concerns about participation in the study from spouse, significant other, or family members;
• Lack of support from primary health care provider;
• Residence too far from the study clinic site such that transportation is a barrier including persons who require transportation assistance provided by the study clinic funds for screening or randomization visits;
• Residence in a nursing home; persons residing in an assisted living or retirement community are eligible if they meet the other criteria;
• Other medical, psychiatric, or behavioral factors that, in the judgment of the site PI or clinician, may interfere with study participation or the ability to follow the study Protocol.
• Couples or significant partners who live together cannot be enrolled or participate simultaneously in the study.

Interventions: DRUG: Angiotensin II Receptor Blockers (ARBs, losartan) and Calcium Channel Blockers (CCB, amlodipine), OTHER: PCP

Conditions: Cognitively Normal Older Adults, Hypertension, Subjective Cognitive Decline, Family History of Dementia, Brain and Nervous System

Keywords: Dementia, Alzheimer's Disease, Cognitive Function, Blood Pressure, Amyloid, Tau

Sites: UT Southwestern

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Ocrelizumab Discontinuation in Relapsing Multiple Sclerosis (AMS05)

Description:

This study is a prospective, multi-center, randomized, double blinded, placebo-controlled study of OCR treatment-discontinuation in patients with early RMS. All eligible participants will be initiated on OCR using the standard approved administration schedule of two 300 mg infusions separated by 14 days (i.e., Days 0 and 14) for a total of 600 mg, followed by 600 mg infusions at Month 6,12, 18, and 24. At Month 24, participants will be randomized (2:1) to one of two Arms with randomized treatment beginning at Month 30: Arm 1: placebo infusions every 6 months; or Arm 2: OCR infusions every 6 months. The treatment period will be for a total of 48 months.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, Manuel.Huichapa@UTSouthwestern.edu

Principal Investigator: Benjamin Greenberg

Gender: ALL

Age: 18 Years to 55 Years old

Phase: PHASE4

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05285891

IRB Number: STU-2022-1218

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Inclusion Criteria:

• Have at least one clinical episode that satisfies McDonald 2017 criteria for early Multiple sclerosis (MS) for up to 2 years post-event with a dissemination in time that can be met clinically, by Magnetic Resonance Imaging (MRI), or based on oligoclonal band (OCB) positivity
• Have a length of disease duration, from first symptom, of ≤ 2 years
• For women of childbearing potential: Agreement to remain abstinent (refrain from heterosexual intercourse) or use effective methods of contraception during the treatment period and for at least 6 months after the last dose of study drug:
• A woman is considered to be of childbearing potential if she is postmenarcheal, has not reached a postmenopausal state (≥12 continuous months of amenorrhea with no identified cause other than menopause), and has not undergone surgical sterilization (removal of ovaries and/or uterus)
• Examples of contraceptive methods include bilateral tubal ligation, male sterilization, established hormonal contraceptives that inhibit ovulation, hormone- releasing intrauterine devices, and copper intrauterine devices
• The reliability of sexual abstinence should be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the participant. Periodic abstinence (e.g., calendar, ovulation, symptothermal, or post ovulation methods) and withdrawal are not acceptable methods of contraception
• Barrier methods must always be supplemented with the use of a spermicide

Exclusion Criteria:

• Inability or unwillingness of a participant to give written informed consent or comply with study protocol
• History of Primary Progressive Multiple Sclerosis (PPMS), Progressive Relapsing Multiple Sclerosis (PRMS), or Secondary Progressive Multiple Sclerosis (SPMS)
• Any metallic material or electronic device in the body, or condition that precludes the participant from undergoing Magnetic resonance imaging (MRI)
• Known presence or history of other neurological disorders, including but not limited to the following:
• Ischemic cerebrovascular disorders, including but not limited to transient ischemic attack, subarachnoid hemorrhage, cerebral thrombosis, cerebral embolism, or cerebral hemorrhage
• Central Nervous System (CNS) or spinal cord tumor, metabolic or infectious cause of myelopathy, genetically inherited progressive CNS disorder, CNS sarcoidosis, or systemic autoimmune disorders potentially causing progressive neurologic disease or affecting ability to perform the study assessments
• Pregnancy or lactation a. Female participants of childbearing potential must have a negative urine pregnancy test at screening
• Any concomitant disease that may require chronic systemic treatment with corticosteroids or immunosuppressants during the course of the study
• Lack of peripheral venous access
• History of severe allergic or anaphylactic reactions to humanized or murine monoclonal antibodies
• Significant, inadequately controlled (e.g. diagnostic evaluations indicated or change in medications warranted) disease, such as cardiovascular (including cardiac arrhythmia), pulmonary (including obstructive pulmonary disease), renal, hepatic, endocrine, and gastrointestinal or any other significant disease that in the opinion of the investigator may preclude participant from participating in the study
• Functional status of NY Heart Association (NYHA) Class III or higher for heart failure at the screening visit
• Known active bacterial, viral, fungal, mycobacterial infection or other infection (including tuberculosis \[TB\] or atypical mycobacterial disease but excluding limited superficial fungal or viral infections of the skin or nails) or any severe episode of infection requiring hospitalization or treatment with Intravenous (IV) antibiotics within 4 weeks prior to baseline visit or oral antibiotics within 2 weeks prior to baseline visit
• Active or chronic infection with Human Immunodeficiency Virus (HIV), syphilis or TB (see laboratory tests below)
• Evidence of past or current hepatitis B or hepatitis C infection, including treated hepatitis B or hepatitis C. Hepatitis B surface antibody following hepatitis B immunization is not considered to be evidence of past infection
• Known active malignancy or active monitoring for recurrence of malignancy, including solid tumors and hematological malignancies, except basal cell, in situ squamous cell carcinoma of the skin, and in situ carcinoma of the cervix or the uterus that have been excised with clear margins
• Substance use disorder, including the recurrent use of alcohol and/or drugs within the past year associated with clinically significant impairment associated with failure to meet major responsibilities at work, school, or home
• Receipt of live or live-attenuated vaccines within 4 weeks prior to baseline
• Contraindications to or severe intolerance of oral or IV corticosteroids, including Intravenous (IV) methylprednisolone administered according to the country label, including:
• Psychosis not controlled by a treatment
• Hypersensitivity to any of the constituents or excipients of the preceding steroids
• Current or prior treatment with the following MS DMTs: fingolimod and other S1P receptor modulators, cladribine, natalizumab, anti-CD20 molecules, alemtuzumab, and chemotherapeutic agents
• Treatment with fumarates within 30 days prior to baseline
• Current or prior treatment with any experimental therapies (e.g., bone marrow transplant), investigational agent, or treatment with any experimental procedure for MS (e.g., treatment for chronic cerebrospinal venous insufficiency)
• Systemic corticosteroid therapy within 4 weeks prior to screening
• Laboratory test results as follows: a. Positive infection screening tests for: i. Hepatitis B surface antigen (HbsAg) or hepatitis B core antibody (HbcAb) ii. Hepatitis C (HCV) antibody, if positive screen for HCV RNA Polymerase Chain Reaction (PCR) iii. Rapid plasma reagin (RPR) iv. HIV v. At or within twelve months of screening: * Positive QuantiFERON(R)-TB Gold test or positive purified protein derivative tuberculin skin test (PPD) (\>5mm induration, regardless of Bacille Calmette Guerin \[BCG\] vaccine administration) unless completion of treatment has been documented for active TB * An indeterminate QuantiFERON(R)-TB Gold test unless followed by a subsequent negative PPD or negative QuantiFERON(R)-TB Gold test as well as a consultation with and clearance by local infectious disease (ID) department b. Levels of serum immunoglobulin G (IgG) \< 3.3g/L c. Estimated glomerular filtration rate (eGFR) \< 60 mL/min/1.73 m2 using Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation d. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT)\>= 2.0 x the upper limit of normal (ULN) e. Platelet count \< 100,000 plt/mcL (\< 100 x 10\^9/L) f. Hemoglobin \< 10 g/dL g. Absolute neutrophil count \< 1.5 × 10⁹/L h. Absolute lymphocyte count \< 1.2 x 10⁹/L
• Past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the investigator, may pose additional risks from participation in the study, may interfere with the participant's ability to comply with study requirements or that may impact the quality or interpretation of the data obtained from the study

Interventions: DRUG: Ocrelizumab, DRUG: Placebo for Ocrelizumab

Conditions: Multiple Sclerosis

Keywords: Ocrelizumab, Multiple sclerosis, Relapse

Sites: UT Southwestern

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Phase 1/2a Study of Belantamab Mafodotin in Relapsed or Refractory AL Amyloidosis

Description:

The goal of this study is to test the safety of drug, Belantamab Mafodotin, and see what effects (good and bad) it has on people who take it and amyloidosis, and to determine the most effective dose of the drug. The study will have 2 phases (parts). The first phase of the study will test different doses of Belantamab Mafodotin. The second phase will test Belantamab Mafodotin at the dose level found to be safe and effective in phase 1

Contact(s):

Call 833-722-6237
canceranswerline@utsouthwestern.edu

Principal Investigator: Larry Anderson

Gender: ALL

Age: 18 Years and over

Phase: PHASE1

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05145816

IRB Number: STU-2021-0952

Show full eligibility criteria

Inclusion Criteria:

• Participants medically diagnosed with relapsed or refractory Amyloid Light Chain Amyloidosis (AL amyloidosis) with one or more line of treatment as below:
• Must have received a proteosome inhibitor, alkylator and anti-cluster of differentiation 38 (CD38) antibody (e.g., daratumumab - for patients who were eligible to receive in newly diagnosed AL Amyloidosis) and autologous stem cell transplant (for transplant eligible candidates). OR
• Failed treatment and/or intolerant/ineligible for above agents NOTE: Patients who fail to achieve Partial Hematological Response or better after 2 cycles of induction therapy for newly diagnosed AL Amyloidosis are also eligible.
• Participant must be over 18 years of age inclusive, at the time of signing the informed consent.
• Participant and Disease Characteristics: Patient must have primary systemic AL amyloidosis, histologically confirmed at the initial diagnosis before initiation of 1st-line treatment by positive Congo red stain with green birefringence on polarized light microscopy, Or characteristic appearance by electron microscopy AND confirmatory AL amyloid typing (mass spectrometry-based proteomic analysis or immunofluorescence).
• Patient must have measurable disease within 28 days prior to registration; serum quantitative immunoglobulins (immunoglobulin G (IgG), immunoglobulin A (IgA), and immunoglobulin M (IgM), serum free kappa and lambda, and serum protein electrophoresis (SPEP) with M-protein quantification must be obtained within 14 days prior to registration.
• Measurable disease of amyloid light chain amyloidosis as defined by at least One of the following:
• Serum M-protein ≥0.5 g/dL by protein electrophoresis (routine serum protein electrophoresis and immunofixation).
• Serum free light chain ≥50 mg/L with an abnormal kappa: lambda ratio or the difference between the involved and uninvolved free light chains (dFLC) ≥50 mg/L.
• One or more organs impacted by AL Amyloidosis according to consensus guidelines below per National Comprehensive Cancer Network (NCCN)Guidelines Version 1.2016: a. Cardiac Involvement i. Mean left ventricular wall thickness on echocardiogram greater than or equal to 12 mm in the absence of hypertension or valvular heart disease, OR N-terminal fragment brain natriuretic protein (NT-pro) brain natriuretic peptide (BNP) greater than 332 ng/mL provided that patient does not have impaired renal function (as defined by calculated creatinine clearance less than 25 mL/min) within 14 days prior to registration, OR prior cardiac biopsy (at time of diagnosis) showing amyloid deposition with past documented or presently noted clinical symptoms and signs supportive of a diagnosis of heart failure in the absence of an alternative explanation for heart failure. b. Non-Cardiac Organ Involvement i. Kidney: albuminuria greater than or equal to 500 mg per day on a 24-hour urine specimen within 35 days prior to registration, OR prior kidney biopsy (at the time of diagnosis) showing amyloid deposition. ii. Liver: hepatomegaly (total liver span \> 15 cm) as demonstrated by computed tomography (CT) or magnetic resonance imaging (MRI) within 35 days prior to registration OR alkaline phosphatase (ALP) greater than 1.5 times the institutional upper limit of normal within 14 days prior to registration, OR prior liver biopsy (at the time of diagnosis) showing amyloid deposition. iii. Gastrointestinal tract: direct biopsy verification with symptoms. iv. Lung: biopsy verifications with symptoms and interstitial radiographic pattern. v. Soft tissue: tongue enlargement, clinical, arthropathy, claudication, presumed vascular amyloid, skin involvement, carpal tunnel syndrome, myopathy by biopsy or pseudohypertrophy.
• Patients must have completed other systemic therapy or investigational drug \> 28 days or five half-lives prior to registration, surgery (other than biopsies) \> 28 days prior to registration, and any autologous stem cell transplant (ASCT) \> 100 days prior to registration.
• Patients must have a complete medical history and physical exam within 14 days prior to registration.
• New York Heart Association (NYHA) Class 1 - 3a which has been clinically stable for 56 days before registration
• Eastern Cooperative Oncology Group (ECOG) performance score 0, 1 or 2
• Left ventricular ejection fraction (LVEF) by echocardiogram (ECHO) \> 35% within 28 days prior to registration.
• Adequate organ system functions within 14 days of registration as defined by the laboratory assessments below: a) Hematologic i) Absolute neutrophil count (ANC): ≥1.0 × 109/ L \* ii) Hemoglobin: ≥8.0 g/dL \* iii) Platelets: ≥50 × 109/L \* b) Hepatic i) Total bilirubin: 1.5 × upper limit of normal (ULN); (Isolated bilirubin ≥1.5 × ULN is acceptable if bilirubin is fractionated, and direct bilirubin is \<35%) ii) Alanine aminotransferase (ALT): ≤2.5 × ULN c) Renal i) Estimated glomerular rate (eGFRª): ≥30 mL/min/1.73 m2 Note: Laboratory results obtained during Screening should be used to determine eligibility criteria. In situations where laboratory results are outside the permitted range, the investigator may re-test the participant and the subsequent within range screening result may be used to confirm eligibility. \* Without growth factor or cell transfusion support for the past 14 days prior to testing, excluding erythropoietin. ª As calculated by Modified Diet in Renal Disease (MDRD) formula (Appendix 4 in Protocol)
• Females of childbearing potential: These participants must have a negative baseline pregnancy test within 72 hours prior to registration; this may be either a serum or urine pregnancy test, with a sensitivity of at least 50 milli-International unit (mIU)/mL; females of childbearing potential must also agree: (1) to have a pregnancy test prior to the start of each treatment cycle and (2) to either commit to continued abstinence from heterosexual intercourse or to use effective contraception while receiving study drug and for at least 4 months after receiving the last dose of study drug; females are considered to be of childbearing potential if they have had menses at any time in the preceding 24 consecutive months; in addition to routine contraceptive methods, effective contraception also includes heterosexual celibacy and surgery intended to prevent pregnancy (or with a side-effect of pregnancy prevention) defined as a hysterectomy, bilateral oophorectomy or bilateral tubal ligation; however, if at any point a previously celibate patient chooses to become heterosexually active during the time period for use of contraceptive measures outlined in the protocol, she is responsible for beginning contraceptive measures.
• Is a woman of child bearing potential (WOCBP) and using a contraceptive method that is highly effective (with a failure rate of \<1% per year), preferably with low user dependency (as described in Appendix 9), during the intervention period and for at least 4 months after the last dose of study intervention and agrees not to donate eggs (ova, oocytes) for the purpose of reproduction during this period. The investigator should evaluate the effectiveness of the contraceptive method in relationship to the first dose of study intervention.
• A WOCBP must have a negative serum pregnancy test (as required by local regulations) within 72 hours before the first dose of study intervention.
• The investigator is responsible for review of medical history, menstrual history, and recent sexual activity to decrease the risk for inclusion of a woman with a nearly undetected pregnancy.
• Non-childbearing potential is defined as follows (by other than medical reasons): i. ≥45 years of age and has not had menses for \>1 year. ii. Patients who have been amenorrhoeic for \<2 years without history of a hysterectomy and oophorectomy must have a follicle stimulating hormone value in the postmenopausal range upon screening evaluation. iii. Post-hysterectomy, post-bilateral oophorectomy, or post-tubal ligation. Documented hysterectomy or oophorectomy must be confirmed with medical records of the actual procedure or confirmed by an ultrasound. Tubal ligation must be confirmed with medical records of the actual procedure.
• Male participants are eligible to participate if they agree to the following during the intervention period and for 6 months after the last dose of study treatment to allow for clearance of any altered sperm:
• Refrain from donating sperm Plus, either:
• be abstinent from heterosexual intercourse as their preferred and usual lifestyle (abstinent on a long term and persistent basis) and agree to remain abstinent Or
• agree to use a barrier method of birth control (e.g., male condom), even if they have undergone a successful vasectomy, and female partner to use an additional highly effective contraceptive method with a failure rate of \<1% per year as when having sexual intercourse with a woman of childbearing potential (including pregnant females).
• Patients with Human Immunodeficiency Virus (HIV) infection are eligible if:
• patients without a history of Acquired Immune Deficiency Syndrome (AIDS)-defining opportunistic infections
• patients with a history of AIDS-defining opportunistic infection may be eligible if they have not had an opportunistic infection within past 12 months.
• Patients on active anti-retroviral therapy are eligible as long as anti-retroviral therapy is established for at least four weeks and have HIV viral load less than 400 copies/ml prior to enrollment.
• Patients with chronic Hepatitis B Virus (HBV) infection or chronic Hepatitis C Virus (HCV) infection or virologically suppressed on HCV treatment are eligible if:
• Hepatitis B surface antigen (HBsAg)-negative, anti-Hemoglobin C (HBc)-positive patients are at lower risk of HBV reactivation compared with HBsAg-positive patients, risk of HBV reactivation should be considered in all patients and if patients can be on anti-HBV prophylaxis prior to initiation of anti-cancer therapy.
• Patients with chronic HBV infection with active disease who meet the criteria for anti HBV therapy should be on a suppressive antiviral therapy prior to initiation of cancer therapy.
• Patients actively on treatment for HCV should have HCV below the limit of quantification before initiation of anti-cancer therapy.
• Patients who are HCV antibody (Ab) positive but HCV Ribonucleic Acid (RNA) negative due to prior treatment or natural resolution of infection are eligible.

Exclusion Criteria:

• Patients previously treated for active symptomatic multiple myeloma.
• Any corneal disease except for mild epithelial punctate keratopathy.
• Patients with known immediate or delayed hypersensitivity reaction or idiosyncratic reactions to belantamab mafodotin or drugs chemically related to belantamab mafodotin, or any of the components of the study treatment.
• Patients eligible for autologous stem cell transplantation (ASCT).
• Evidence of significant cardiovascular condition as specified below:
• N-terminal-prohormone of brain natriuretic peptide (NT-proBNP) ≥ 8500ng/L within 14 days of registration.
• New York Heart Association (NYHA) classification IIIB (3b) through IV (4) heart failure
• Heart failure that in the opinion of the investigator is on the basis of ischemic heart disease (e.g., prior myocardial infarction with documented history of cardiac enzyme elevation and electrocardiogram (ECG) changes) or uncorrected valvular disease and not primarily due to AL amyloid cardiomyopathy
• Unstable heart failure defined as emergency hospitalization for worsening, or decompensated heart failure, or syncopal episode within 1 month of screening
• Subjects with a history of sustained ventricular tachycardia or aborted ventricular fibrillation or with a history of atrioventricular nodal or sinoatrial (SA) nodal dysfunction for which a pacemaker/implantable cardioverter-defibrillator (ICD) is indicated but not placed (Subjects who do have a pacemaker/ICD are allowed on study)
• Interval from the Q wave on the ECG to point T using Fredericia's formula (QTcF) \> 500 msec. Subjects who have a pacemaker may be included regardless of calculated QTc interval
• Symptomatic, clinically significant autonomic neuropathy which the Investigator feels will preclude administration of study treatment
• Acute coronary syndrome, or any form of coronary revascularization procedure including coronary artery bypass grafting (CABG), within 6 months of screening
• Prior solid organ transplant, or anticipated to undergo solid organ transplantation, or requiring left ventricular assist device (LVAD) implantation, during the course of the study
• Stroke within 6 months of screening, or transient ischemic attack (TIA) within 3 months of screening
• Evidence of current clinically significant uncontrolled arrhythmias, including clinically significant ECG abnormalities such as 2nd degree (Mobitz Type II) or 3rd degree atrioventricular (AV) block
• History of myocardial infarction, acute coronary syndromes (including unstable angina), coronary angioplasty, or stenting or bypass grafting within three (3) months of Screening
• Uncontrolled hypertension
• Prior history of malignancy with the exception of the following: adequately treated basal cell or squamous cell skin cancer, curatively treated non-melanoma skin cancer, in situ cervical cancer, adequately treated stage I or II cancer from which the patient is currently in complete remission, or any other cancer from which the patient has been disease free for at least two years.
• Presence of any comorbid or uncontrolled medical condition (e.g., diabetes mellitus or uncontrolled hypertension) at screening, which in the opinion of the investigator would increase the potential risk to the subject.
• Unwillingness or inability to follow the procedures outlined in the protocol.
• Received an investigational drug (including investigational vaccines) or used an invasive investigational medical device within 4 weeks or five half-lives, whichever is shorter, before Cycle 1 Day 1.
• Participant must not use contact lenses while participating in this study.
• Participant must not have had major surgery ≤ 4 weeks prior to initiating study treatment.
• Participant must not have any evidence of active mucosal or internal bleeding.
• Participant must not have any serious and/or unstable pre-existing medical, psychiatric disorder, or other conditions (including lab abnormalities) that could interfere with participant's safety, obtaining informed consent or compliance to the study procedures.
• Participants must not be pregnant or lactating.
• Participant must not be simultaneously enrolled in any interventional clinical trial.
• Participant must not have an active infection requiring treatment.
• Participant must not have current unstable liver or biliary disease defined by the presence of ascites, encephalopathy, coagulopathy, hypoalbuminemia, esophageal or gastric varices, persistent jaundice, or cirrhosis. Note: Stable non-cirrhotic chronic liver disease (including Gilbert's syndrome or asymptomatic gallstones) or hepatobiliary involvement of malignancy is acceptable if otherwise meets entry criteria.

Interventions: DRUG: Belantamab mafodotin 2.5 mg/kg (8 weeks), DRUG: Belantamab mafodotin 1.9 mg/kg (8 weeks), DRUG: Belantamab mafodotin 1.4 mg/kg (12 weeks), DRUG: Belantamab mafodotin 1.9 mg/kg (12 weeks), DRUG: Belantamab mafodotin every 4 weeks, 6 weeks,8 weeks, or 12 weeks as determined by Part 1 recommended dosages, DRUG: Belantamab mafodotin 1.0 mg/kg (12 weeks)

Conditions: AL Amyloidosis, Amyloidosis, Multiple Myeloma

Sites: UT Southwestern

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A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (HOPE-3)

Description:

HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy (DMD) and impaired skeletal muscle function. Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during the first 12-months of the study. All participants will be eligible to receive 4 doses of CAP-1002 for an additional 12 months as part of an open-label extended assessment period.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, Tammy.Ramm@UTSouthwestern.edu

Principal Investigator: Susan Iannaccone

Gender: MALE

Age: 10 Years and over

Phase: PHASE3

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05126758

IRB Number: STU-2022-0124

Show full eligibility criteria

Inclusion Criteria:

• Male subjects at least 10 years of age at time of consent who are willing and able to provide informed consent to participate in the trial if ≥ 18 years of age or assent with parental or guardian informed consent if \< 18 years of age. If a third-party caregiver is involved, they must provide informed consent.
• Diagnosis of DMD based on clinical and phenotypic manifestations consistent with DMD (e.g., family history of DMD, elevated creatine kinase, dystrophin muscle biopsy, calf pseudohypertrophy, history of Gowers' sign, and gait impairment before 7 years of age) as confirmed by the Investigator.
• Confirmatory genetic testing performed to have reached a diagnosis of DMD at any time in the past or currently performed at a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory or equivalent.
• Performance of the Upper Limb test (PUL) entry item scores 2-6 and total PUL score less than or equal to 40. For Cohort A only: enrollment of patients with PUL entry score 6, Exon 44 skipping amenable, and/or Exon 3 through 7 deletions will be capped at no more than 10% of the total study population (approximately 6 patients with these characteristics).
• Reduced ability to walk/run (if ambulatory): subjects must take more than 10 seconds for the 10-meter walk/run (i.e., velocity \< 1 meter/second).
• If non-ambulatory, loss of independent ambulation between 10th and 18th year birthday (standing unassisted or ability to take, at most, several steps independently is not considered ambulation). Subjects who are considered non-ambulatory between the ages of 9 and10 may be enrolled with prior approval from the sponsor.
• Receiving standard of care therapy at an experienced, multidisciplinary DMD center as evidenced by regular cardiac and pulmonary monitoring, systemic glucocorticoid treatment, and at-home range of motion exercises.
• Treatment with systemic glucocorticoids for at least 12 months and at a stable dose at least 6 months prior to study participation, except for either weight-based dose adjustment or a decrease in steroid dose of ≤ 10% for toxicity. For patients on chronic deflazacort, treatment with an equivalent dose of prednisone or prednisolone for a period of ≤ 30 days to bridge lack of availability of deflazacort during the 6 months prior to randomization is acceptable.
• Current and up-to-date immunizations according to children and adolescent Centers for Disease Control and Prevention immunization schedule at the discretion of the Investigator.
• Adequate venous access for parenteral IP infusions and routine blood collection.
• Assessed by the Investigator as willing and able to comply with the requirements of the trial.
• Sexually active subjects and their partners who are fertile must agree to use effective method(s) of contraception.

Exclusion Criteria:

• Left ventricular ejection fraction (LVEF) less than or equal to 35% prior to randomization.
• Elbow-flexion contractures \> 30° in both extremities.
• Body mass index (BMI) \> 45.
• Percent predicted forced vital capacity (FVC%) \< 35% within 6 months prior to randomization.
• Inability to perform consistent PUL 2.0 measurement within ± 2 points without shoulder domain or within ± 3 points with shoulder domain during paired testing at screening.
• Risk of near-term respiratory decompensation in the judgment of the Investigator, or the need for initiation of day and night non-invasive ventilator support as defined by serum bicarbonate ≥ 29 mmol/L at screening.
• History of non DMD-related chronic respiratory disease requiring ongoing or intermittent treatment, including, but not limited to, asthma, bronchitis, and tuberculosis.
• Acute respiratory illness within 30 days prior to screening and during screening.
• Initiation of nocturnal non-invasive ventilation within 30 days prior to screening.
• Planned or anticipated thoracic or spinal surgery within the 6 months following randomization.
• Planned or anticipated lower extremity surgery within the 6 months following randomization, if ambulatory.
• Known hypersensitivity to dimethyl sulfoxide (DMSO) or bovine products.
• Initiation of treatment with metformin or insulin within 3 months prior to randomization.
• Initiation of treatment with an FDA-approved exon skipping therapy for the treatment of DMD and/or non-weight based adjustments within 12 months prior to randomization.
• Treatment with human growth hormone within 3 months prior to randomization, unless on a stable dose allowing for weight-based dose adjustments (as determined by the site Investigator) for at least 24 months prior to randomization.
• Treatment with a cell therapy product within 12 months prior to randomization; any prior exposure to CAP-1002 will be excluded.
• Treatment with an investigational product within 6 months prior to randomization.
• History, or current use, of drugs or alcohol that could impair the ability to comply with participation in the trial.
• Inability to comply with the investigational plan and follow-up visit schedule for any reason, in the judgment of the investigator.
• Inability to undergo a cardiac MRI. For Cohort B Only - Subjects with a known hypersensitivity to gadolinium may forgo the LGE assessment but must complete a cardiac MRI without contrast. For Cohort B Only - Subjects who are unable to tolerate gadolinium due to renal insufficiency as measured by an estimated Glomerular Filtration Rate (eGFR) less than 60 mL/min/1.73 m2 may forgo the LGE assessment but must complete a cardiac MRI without contrast.
• For Cohort B: Subjects with PUL entry score 6, Exon 44 skipping amenable, or Exon 3 through 7 deletions are excluded from participation.

Interventions: BIOLOGICAL: CAP-1002, BIOLOGICAL: Placebo

Conditions: Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases, Neuromuscular Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn, Nervous System Diseases

Keywords: Duchenne Muscular Dystrophy, Cell Therapy, Performance of the Upper Limb, Ambulatory, Non-Ambulatory

Sites: Children’s Health

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Testing the Addition of the Chemotherapy Drug Lomustine (Gleostine) to the Usual Treatment (Temozolomide and Radiation Therapy) for Newly Diagnosed MGMT Methylated Glioblastoma

Description:

This phase III trial compares the effect of adding lomustine to standard chemotherapy with temozolomide and radiation therapy versus temozolomide and radiation therapy alone in shrinking or stabilizing newly diagnosed MGMT methylated glioblastoma. MGMT methylated tumors are more likely to respond to temozolomide chemotherapy. Temozolomide is in a class of medications called alkylating agents. It works by damaging the cell's DNA and may kill tumor cells and slow down or stop tumor growth. Lomustine is a chemotherapy drug and in a class of medications called alkylating agents. It damages the cell's DNA and may kill tumor cells. Radiation therapy uses high energy x-ray photons to kill tumor cells and shrink tumors. Adding lomustine to standard chemotherapy with temozolomide and radiation therapy may shrink or stabilize glioblastoma.

Contact(s):

Call 833-722-6237
canceranswerline@utsouthwestern.edu

Principal Investigator: Nawal Shaikh

Gender: ALL

Age: 18 Years to 70 Years old

Phase: PHASE3

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05095376

IRB Number: STU-2022-0465

Show full eligibility criteria

Inclusion Criteria:

* STEP 1 REGISTRATION: No known IDH mutation. (If tested before step 1 registration, patients known to have IDH mutation in the tumor on local or other testing are ineligible and should not be registered) * STEP 1 REGISTRATION: Availability of formalin-fixed paraffin-embedded (FFPE) tumor tissue block and hematoxylin and eosin (H\&E) stained slide to be sent for central pathology review for confirmation of histology and MGMT promoter methylation status. Note that tissue for central pathology review and central MGMT assessment must be received by the New York University (NYU) Center for Biospecimen Research and Development (CBRD) on or before postoperative calendar day 30. If tissue cannot be received by postoperative calendar day 30, then patients may NOT enroll on this trial as central pathology review will not be complete in time for the patient to start treatment no later than 8 weeks following surgery. Results of central pathology review and central MGMT analysis will generally be completed within 10 business days of receipt of tissue. Results will be entered by the central lab directly into Rave. Note: In the event of an additional tumor resection(s), tissue must be received within 30 days of the most recent resection and the latest resection must have been performed within 30 days after the initial resection. Surgical resection is required; stereotactic biopsy alone is not allowed because it will not provide sufficient tissue for MGMT analysis * STEP 1 REGISTRATION: Willing to use highly effective method of contraception for participants of childbearing potential (participants who may become pregnant or who may impregnate a partner) during therapy and for 6 months after completing treatment; this inclusion is necessary because the treatment in this study may be significantly teratogenic * STEP 1 REGISTRATION: The patient or a legally authorized representative must provide study-specific informed consent prior to study entry and, for patients treated in the United States (U.S.), authorization permitting release of personal health information * STEP 2 REGISTRATION: Histopathologically proven diagnosis of glioblastoma (or gliosarcoma as a subtype of glioblastoma) confirmed by central pathology review * STEP 2 REGISTRATION: MGMT promoter with methylation confirmed by central pathology review. Note: Patients with tissue that is insufficient or inadequate for analysis, fails MGMT testing, or has indeterminate or unmethylated MGMT promoter are excluded * Note: Any MGMT result other than methylated would require step 2 registration to be reported as a "central review failure" * STEP 2 REGISTRATION: Contrast-enhanced brain MRI performed either after surgery or prior to step 2 registration * STEP 2 REGISTRATION: IDH mutation testing by at least one method (such as immunohistochemistry for IDH1 R132H) must be performed as part of standard of care and no mutation must be found (i.e IDH wildtype). (If a mutation is identified then the patient will be ineligible and must be registered as ineligible at step 2.) * STEP 2 REGISTRATION: History/physical examination within 28 days prior to step 2 registration * STEP 2 REGISTRATION: Karnofsky performance status (KPS) \>= 70 within 28 days prior to step 2 registration * STEP 2 REGISTRATION: Neurologic function assessment within 28 days prior to step 2 registration * STEP 2 REGISTRATION: Age 18-70 years * STEP 2 REGISTRATION: Hemoglobin \>= 10 g/dl (Note: the use of transfusion or other intervention to achieve hemoglobin \[Hgb\] \>= 10.0 g/dl is acceptable) (Within 14 days prior to step 2 registration) * STEP 2 REGISTRATION: Leukocytes \>= 2,000/mm\^3 (Within 14 days prior to step 2 registration) * STEP 2 REGISTRATION: Absolute neutrophil count \>= 1,500/mm\^3 (Within 14 days prior to step 2 registration) * STEP 2 REGISTRATION: Platelets \>= 100,000/mm\^3 (Within 14 days prior to step 2 registration) * STEP 2 REGISTRATION: Total bilirubin =\< 1.5 x institutional/lab upper limit of normal (ULN) (Within 14 days prior to step 2 registration) * STEP 2 REGISTRATION: Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase \[SGOT\]) =\< 2.5 x ULN (Within 14 days prior to step 2 registration) * STEP 2 REGISTRATION: Alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase \[SGPT\]) =\< 2.5 x ULN (Within 14 days prior to step 2 registration) * STEP 2 REGISTRATION: Serum creatinine =\< 1.5 x ULN OR creatinine clearance (CrCl) \>= 50 mL/min (if using the Cockcroft-Gault formula) (Within 14 days prior to step 2 registration) * STEP 2 REGISTRATION: For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated * Note: Known positive test for hepatitis B virus surface antigen (HBV sAg) indicating acute or chronic infection would make the patient ineligible unless the viral load becomes undetectable on suppressive therapy. Patients who are immune to hepatitis B (anti-hepatitis B surface antibody positive) are eligible (e.g. patients immunized against hepatitis B) * STEP 2 REGISTRATION: For patients with a history of hepatitis C virus (HCV) infection must have been treated and cured. For patients with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load * Note: Known positive test for hepatitis C virus ribonucleic acid (HCV ribonucleic acid \[RNA\]) indicating acute or chronic infection would make the patient ineligible unless the viral load becomes undetectable on suppressive therapy * STEP 2 REGISTRATION: Known human immunodeficiency virus (HIV) infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months prior to step 2 registration are eligible for this trial. Testing is not required for entry into protocol * STEP 2 REGISTRATION: Negative serum or urine pregnancy test (in persons of childbearing potential) within 14 days prior to step 2 registration * Childbearing potential is defined as any person who has experienced menarche and who has not undergone surgical sterilization (hysterectomy or bilateral oophorectomy) or who is not postmenopausal

Exclusion Criteria:

* STEP 2 REGISTRATION: Prior therapy for tumor except for resection or prior laser interstitial thermal therapy (LITT). For example, prior chemotherapy, immunotherapy, or targeted therapy for GBM or lower grade glioma is disallowed (including but not limited to temozolomide, lomustine, bevacizumab, any viral therapy, ipilimumab or other CTLA-4 antibody, PD-1 antibody, CD-137 agonist, CD40 antibody, PDL-1 or 2 antibody, vaccine therapy, polio or similar viral injection as treatment for the tumor, and/or any other antibody or drug specifically targeting T-cell co-stimulation or immune checkpoint pathways) as is Gliadel wafer, radiotherapy, radiosurgery, vaccine or other immunotherapy, brachytherapy, or convection enhanced delivery * Note: 5-aminolevulinic acid (ALA)-mediated fluorescent guided resection (FGR) photodynamic therapy (PDT) or fluorescein administered prior to/during surgery to aid resection is not exclusionary and is not considered a chemotherapy or intracerebral agent. Prior laser interstitial thermal therapy (LITT) is allowed * STEP 2 REGISTRATION: Current or planned treatment with any other investigational agents for the study cancer * STEP 2 REGISTRATION: Definitive clinical or radiologic evidence of metastatic disease outside the brain * STEP 2 REGISTRATION: Prior invasive malignancy (except non-melanomatous skin cancer, cervical cancer in situ and melanoma in situ) unless disease free for a minimum of 2 years * STEP 2 REGISTRATION: Prior radiotherapy to the head or neck that would result in overlap of radiation therapy fields * STEP 2 REGISTRATION: Pregnancy and individuals unwilling to discontinue nursing due to the potential teratogenic effects and potential risk for adverse events in nursing infants * STEP 2 REGISTRATION: History of allergic reactions attributed to compounds of similar chemical or biologic composition to temozolomide or lomustine * STEP 2 REGISTRATION: History of pulmonary fibrosis * STEP 2 REGISTRATION: Uncontrolled intercurrent illness including, but not limited to: * Ongoing or active infection requiring intravenous (IV) antibiotics, IV antiviral, or IV antifungal treatment * Symptomatic congestive heart failure, defined as New York Heart Association Functional Classification III/IV (Note: Patients with known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association Functional Classification) * Unstable angina pectoris within 6 months prior to Step 2 registration * Uncontrolled cardiac arrhythmia * Psychiatric illness/social situations that would limit compliance with study requirements * STEP 2 REGISTRATION: No evidence of diffuse leptomeningeal disease that requires whole brain irradiation

Interventions: DRUG: Lomustine, PROCEDURE: Magnetic Resonance Imaging, RADIATION: Photon Beam Radiation Therapy, OTHER: Questionnaire Administration, DRUG: Temozolomide

Conditions: Glioblastoma, Gliosarcoma, Brain and Nervous System

Sites: UT Southwestern; Parkland Health & Hospital System

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