UTSW - Study Finder

Open-label Extension Study to Evaluate Metreleptin in Patients with Partial Lipodystrophy

Contact(s):

studyfinder@utsouthwestern.edu

Principal Investigator:

Gender: ALL

Age: 13 Years to old

Phase: PHASE3

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT06679270

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Inclusion Criteria:

• Age ≥13 years of age, inclusive, at the time of signing the informed consent form (ICF).
• Subjects must have completed the Parent study APG-20 and, in the opinion of the Investigator and Sponsor, have been compliant with study procedures through Parent study Month 12 visit.
• Negative pregnancy test (urine or serum) for female subjects of childbearing potential
• Female subjects must be postmenopausal (defined as cessation of menses for at least 1 year), surgically sterile (hysterectomy, bilateral oophorectomy, or tubal ligation), or willing to use a highly effective method of contraception (such methods include combined \[estrogen and progestogen containing\] hormonal contraception associated with inhibition of ovulation: oral/intravaginal; transdermal/progestogen-only hormonal contraception associated with inhibition of ovulation: oral/injectable; implantable/intrauterine device \[IUD\]/intrauterine hormone-releasing system \[IUS\]/bilateral tubal occlusion/vasectomized partner/sexual abstinence) for the duration of the study (from the time they sign an ICF, until 4 weeks after the last dose of study treatment). Hormonal contraception alone (including oral, injectable, transdermal, and implantable) is not acceptable; an additional barrier method must be used. Intravaginal hormonal contraception or IUS alone are allowed per Investigator's discretion. Subjects on oral contraceptives will not be required to discontinue medication. Subjects will not be permitted to commence oral contraceptives while taking study treatment during the study.
• Male subjects must be surgically sterile or willing to use an acceptable method of contraception for the duration of the study (from the time they sign an ICF), until 4 weeks after the last dose of study treatment. An acceptable method of contraception would be a barrier method, such as condoms, restraining from having sex, or a partner using the approved methods of contraception for female subjects as per Inclusion Criteria #4.
• Subjects who are blood/egg/sperm donors should be willing to halt donations during the study and for 4 weeks following their last dose of study treatment.
• Subjects who are willing to provide informed consent/assent prior to any study-specific procedures. If a minor, the subject has a parent or legal guardian able to read, understand, and sign the ICF and/or a Child Assent Form (if applicable), communicate with the Investigator, and understand and comply with the protocol requirements. Adolescent subjects must also read and understand the Child Assent Form.
• Subjects who are willing to follow the dietary restrictions recommended by the Investigator.

Exclusion Criteria:

• Severe hypersensitivity reactions to the study treatment of the Parent study APG-20.
• Known to have tested positive for human immunodeficiency virus (HIV) or known to be diagnosed with HIV-related LD. Positive HIV test in countries requiring HIV testing.
• Are immunocompromised or receiving immunomodulatory drugs.
• Estimated glomerular filtration rate (eGFR) \<30 mL/min/1.73m2 calculated by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) for subjects ≥18 years of age and by Bedside Schwartz for subjects \<18 years of age.
• Diagnosis of clinically significant hematological abnormalities (including but not limited to clinically significant leukopenia, neutropenia, bone marrow abnormalities, leukemia or lymphoma, or clinically significant pathological lymphadenopathy).
• Malignancy that is ongoing/not in remission or that currently requires or has required active treatment within the past year (with the exception of basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or carcinoma in situ \[e.g., breast carcinoma, cervical cancer in situ\] that have undergone potentially curative therapy).
• For females only: currently pregnant (confirmed with a positive pregnancy test) or breastfeeding.
• Any condition where, in the opinion of the Investigator, participation in this study may pose a significant risk to the subject.

Interventions: DRUG: Metreleptin

Conditions: Familial Partial Lipodystrophy

Keywords: Metreleptin, familial partial lipodystrophy

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Infant Formula in Infants and Children With Cow's Milk Allergy

Contact(s):

studyfinder@utsouthwestern.edu

Principal Investigator:

Gender: ALL

Age: 3 Months to 12 Years old

Phase: NA

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT06456541

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Inclusion Criteria:

* Participant's parent(s) is/are willing for their child to undergo confirmatory testing or must have had at least one of the following within 6 months of enrollment:
• Physician diagnosis of IgE-mediated CMA, according to the participant's clinical history (e.g. gastrointestinal symptoms, hives, respiratory symptoms or angioedema) AND, detectable serum milk-specific IgE \>0.7 kUᴀ/L or positive skin prick test wheal ≥5 mm;
• Documentation of milk-specific serum IgE \>15 kUᴀ/L or \> 5 kUᴀ/L if younger than 1 year;
• Documented cow's milk skin prick test wheal \>10mm;
• Physician-supervised oral food challenge that elicited immediate, objective, allergic symptoms. * Participant's parent(s) agree for their child to stop oral steroid use within 14 days and antihistamine use within 7 days of confirmation of diagnosis and food challenges. * Participant had followed a strict cow's milk protein-free diet for at least 2 weeks prior to enrollment. * Parent(s) confirm their intention not to administer any products containing cow's milk protein during the study. * Participant is between 3 months and 12 years of age at enrollment. * Participant's parent(s) has voluntarily signed and dated an Informed Consent Form (ICF), approved by an Independent Ethics Committee/Institutional Review Board (IEC/IRB) and provided Health Insurance Portability and Accountability Act (HIPAA) (or other applicable privacy regulation) authorization prior to any participation in the study.

Exclusion Criteria:

* Participant is partially or exclusively breastfed at the time of enrollment. * Participant is consuming an amino acid-based formula due to failure on an extensively hydrolyzed formula. * Significant chronic medical diseases including major chromosomal or congenital anomalies, gastrointestinal diseases, or abnormalities other than CMA, immunodeficiencies, unstable asthma, asthma treated with biologics (omalizumab or other monoclonal antibody), FPIES, eosinophilic esophagitis, and severe uncontrolled eczema. * Previous severe anaphylactic reaction to cow's milk within the last two years., * An adverse medical history or current condition that is thought by the investigator to have potential for effects on tolerance or the hypoallergenicity test. * Participant has an allergy or intolerance to any ingredient in the study product, as reported by the parent. * Participant is consuming baked milk products.

Interventions: OTHER: Experimental Extensively Hydrolyzed Formula, OTHER: Placebo Extensively Hydrolyzed Formula

Conditions: Cow's Milk Allergy

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Global Study of Del-desiran for the Treatment of DM1 (HARBOR)

Description:

A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1

Contact(s):

studyfinder@utsouthwestern.edu

Principal Investigator:

Gender: ALL

Age: 16 Years to 65 Years old

Phase: PHASE3

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT06411288

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Interventions: DRUG: AOC 1001 (del-desiran), DRUG: Placebo

Conditions: DM1, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonia, Myotonic Dystrophy Type 1 (DM1), Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, Steinert, Myotonic Muscular Dystrophy

Keywords: DM1, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonia, Myotonic Dystrophy Type 1 (DM1), Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, Steinert, Myotonic Muscular Dystrophy, HARBOR, Avidity Biosciences, Avidity, AOC 1001, Del-desiran, Delpacibart etedesiran

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A Culturally-Tailored Mobile Health and Social Media Physical Activity Intervention for Improving Physical Activity in Hispanic or Latino/Latina Adolescent and Young Adult Childhood Cancer Survivors, Walking Juntos Study

Contact(s):

studyfinder@utsouthwestern.edu

Principal Investigator:

Gender: ALL

Age: 15 Years to 20 Years old

Phase: PHASE2

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT06410209

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Inclusion Criteria:

* Patient must be ≥ 15 years and \< 21 years at the time of enrollment * First diagnosis of malignant neoplasm (International Classification of Diseases for Oncology \[ICD-O\] behavior code of "3") in first and continuous remission at the time of enrollment * Curative cancer treatment must have included chemotherapy (including cellular therapy) and/or radiation (including radioactive iodine). Note: Children's Oncology Group (COG) therapeutic trial participation is not required * Completed all chemotherapy and/or radiation therapy in the last 3-36 months. This includes completion of all oral (e.g., tyrosine kinase inhibitors) and/or maintenance chemotherapy * Self-report of \< 420 minutes of moderate-to-vigorous physical activity per week as assessed and documented via the study-specific Physical Activity Worksheet. Note: See the case report forms packet on the COG study web page for the study specific Physical Activity Worksheet * Ambulatory and no known medical contraindications to increasing physical activity * No known significant physical or cognitive impairment that would prevent use of the electronic devices used for the protocol intervention (e.g., Fitbit, smartphone, tablet, or computer) * Able to read and write Spanish or English * Self-identify as Hispanic, Latino/Latina/Latinx

Exclusion Criteria:

* Patients with previous allogeneic hematopoietic stem cell transplant (HSCT) are excluded. Note: Patients with previous autologous HSCT, chimeric antigen receptor T-cell (CAR T-cell) therapy, and other cellular cancer therapies can participate as long as all other eligibility criteria are satisfied * Post-menarchal female patients who are pregnant or planning to become pregnant in the next year are excluded. Note: Pregnancy status can be established by clinical history with patient. Post-menarchal female patients are eligible as long as they agree to use an effective contraceptive method (including abstinence) during study participation * Participants who were enrolled in ALTE2031 (Step by Step) cannot enroll in ALTE2321. Participants who were enrolled in ALTE2321 Stage 1 (cultural tailoring) cannot enroll to participate in Stage 2 (RCT) * All patients and/or their parents or legal guardians must sign a written informed consent. Note: Informed consent may be obtained electronically/online if allowed by local site policy and institutional review board (IRB)/Research Ethics Board (REB) of record * All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met

Interventions: OTHER: Goal Setting, OTHER: Interview, OTHER: Interview, OTHER: Media Intervention, OTHER: Medical Device Usage and Evaluation, OTHER: Medical Device Usage and Evaluation, OTHER: Questionnaire Administration, OTHER: Reward, OTHER: Text Message-Based Navigation Intervention, OTHER: Text Message-Based Navigation Intervention

Conditions: Hematopoietic and Lymphatic System Neoplasm, Malignant Solid Neoplasm

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Screening Emotions in Adolescents at the Hospital for mTBI (SEARCH-mTBI)

Description:

The goal of this observational study is to develop and validate a clinical tool to predict which adolescents aged 11 to less than 18 years of age with mild traumatic brain injury (mTBI) are at an increased risk for developing significant new or worsening mental health conditions. The main aims the study wish to answer are: * Does the adolescent have new or worsening depression or anxiety defined as a change from their previous medical history using self-reported questionnaires at either one or three months post-injury? * Does the adolescent have unmet mental health care needs, defined as not receiving any mental or behavior health care in patients with new or worsening anxiety or depression as defined by the self reported questionnaires? Participants will be enrolled after being diagnosed in the emergency department (ED) with an mTBI. During the ED visit, the child's parent/caregiver and the adolescent will complete several questionnaires related to mental health which include tools to measure anxiety and depression. Participants will be asked to complete these questionnaires again at 1 month and 3 months post enrollment.

Contact(s):

Aja Bayo, MSc aja.bayo@utsouthwestern.edu

Principal Investigator:

Gender: ALL

Age: 11 Years to 17 Years old

Phase:

Healthy Volunteers:

System ID: NCT06370520

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Interventions: BEHAVIORAL: Validated Questionnaires, BEHAVIORAL: Questionnaires, OTHER: Clinician / Medical Record Variables

Conditions: Brain Injury Traumatic Mild, Brain Injuries, Brain Injuries, Acute, Head Injury With Intracranial Hemorrhage, Head Injury Trauma, Brain Injury Traumatic Focal With Loss of Consciousness, Skull Fractures, Diffuse Axonal Injury, Intracranial Hemorrhages, Head Injury

Keywords: Child, Concussion, Wounds and Injuries, Brain Injuries, Head Injuries Trauma, Loss of Consciousness

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A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)

Description:

This is a 10-year multi-center, prospective, longitudinal, single arm study evaluating immunologic, inflammatory and laboratory parameters associated with long-term Palynziq treatment in subjects with phenylketonuria (PKU) in the United States (US). Subjects in the US for whom a clinical decision has been made that they will receive pegvaliase to treat their PKU within 30 days following the date of enrollment in Study 165-501 (incident-users) or who have previously started treatment with pegvaliase at the date of enrollment in Study 165-501 (prevalent-users) are eligible for participation in Study 165-503.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, Juana.Luevano@UTSouthwestern.edu

Principal Investigator: Markey McNutt

Gender: ALL

Age:

Phase:

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT06305234

IRB Number: STU-2023-0625

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Interventions: DRUG: Pegvaliase

Conditions: Phenylketonuria (PKU)

Keywords: PKU, Phenylketonuria, Palynziq, pegvaliase, observational, safety study, immunogenicity assessment, inflammatory assessment

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):

studyfinder@utsouthwestern.edu

Principal Investigator:

Gender: ALL

Age: to 30 Years old

Phase: PHASE3

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT06172296

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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 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 derived from the Schwartz formula for estimating glomerular filtration rate (GFR) utilizing child length and stature data published by the CDC or * a 24-hour urine creatinine clearance ≥ 70 mL/min/1.73 m\^2 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, 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, RADIATION: Radiation Therapy, PROCEDURE: Radionuclide Imaging, OTHER: Survey Administration, DRUG: Temozolomide, DRUG: Thiotepa, DRUG: Topotecan, PROCEDURE: Tumor Resection, DRUG: Vincristine

Conditions: Ganglioneuroblastoma, Nodular, Neuroblastoma

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Safety and Efficacy of TSHA-102 in Pediatric Females with Rett Syndrome (REVEAL Pediatric Study)

Description:

The REVEAL Pediatric Study is a multi-center, Phase 1/2 open-label, dose-escalation and dose-expansion study of TSHA-102, an investigational gene therapy, in pediatric females with Rett Syndrome. The safety, tolerability, and preliminary efficacy of two dose levels will be evaluated. The study duration is up to 6 years.

Contact(s):

studyfinder@utsouthwestern.edu

Principal Investigator:

Gender: FEMALE

Age: 5 Years to 8 Years old

Phase: PHASE1

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT06152237

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Interventions: GENETIC: TSHA-102

Conditions: Rett Syndrome

Keywords: Rett Syndrome, Neurodevelopmental Disorder, MECP2

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A Study to Assess the Efficacy, Safety and Pharmacokinetics of Debio 4326 in Pediatric Participants Receiving Gonadotropin-Releasing Hormone Agonist Therapy for Central Precocious Puberty (LIBELULA)

Description:

The primary objective of this study is to evaluate the efficacy of Debio 4326 in suppressing serum luteinizing hormone (LH) to prepubertal levels 52 weeks after the first Debio 4326 injection in pediatric participants receiving gonadotropin-releasing hormone agonist (GnRHa) therapy for central precocious puberty (CPP).

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, yazmin.molina@childrens.com

Principal Investigator: Perrin White

Gender: ALL

Age: 5 Years to 8 Years old

Phase: PHASE3

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT06129539

IRB Number: STU-2023-1195

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Inclusion Criteria:

• Diagnosis of central precocious puberty and currently receiving GnRHa therapy.
• Onset of development of sex characteristics (i.e., breast development in girls or testicular enlargement in boys according to the Tanner method) before the age of 8 years in girls and 9 years in boys.
• Initially, only participants aged (a) 5 to 8 years inclusive (i.e., \<9 years) are eligible. The Sponsor will determine based on the recommendation of the DMC following the interim analysis whether participants aged 2 to 4 years inclusive (i.e., \<5 years) and/or 9 to 10 years inclusive (i.e., \<11 years) may be recruited.
• Participant to receive at least 1 year of GnRHa therapy from study treatment start.
• Start of initial GnRHa therapy no later than 18 months after onset of the first signs of Central precocious puberty (CPP).
• Difference between bone age (Greulich and Pyle method) and chronological age of ≥1 year based on historical values at the initiation of the GnRHa therapy.
• Pubertal-type LH response following a GnRH/GnRHa stimulation test, or random non-stimulated serum (if considered local standard of care), based on historical values prior to the initiation of GnRHa therapy.
• Clinical evidence of puberty, defined as Tanner Staging ≥2 for breast development for girls and testicular volume ≥4 mL (cc) for boys, prior to the initiation of GnRHa therapy.

Exclusion Criteria:

• Gonadotropin-independent (peripheral) precocious puberty: gonadotropin-independent gonadal or adrenal sex steroid secretion.
• Non-progressing, isolated premature thelarche prior to the initial GnRHa therapy.
• Presence of an unstable intracranial tumor or an intracranial tumor potentially requiring neurosurgery or cerebral irradiation. Participants with hamartomas not requiring surgery are eligible.
• Any other condition or chronic illness possibly interfering with growth (e.g., renal failure, diabetes, moderate to severe scoliosis, previously treated intracranial tumor).
• Other than GnRHa therapy, any ongoing treatment with a potential effect on serum levels of gonadotropins or sex steroids, or possibly interfering with growth.
• Prior or current therapy with medroxyprogesterone acetate, growth hormone, or Insulin-like growth factor-1 (IGF-1).
• Diagnosis of short stature, i.e., more than 2.25 standard deviations (SD) below the mean height-for-age.
• Known history of seizures, epilepsy, and/or central nervous system disorders that may have been associated with seizures or convulsions.
• Prior (within 2 months of study treatment start) or current use of medications that have been associated with seizures or convulsions.
• Use of anticoagulants (heparin or coumarin derivatives).

Interventions: DRUG: Debio 4326

Conditions: Central Precocious Puberty

Sites: Children’s Health

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A Safety Assessment of Oral Letermovir in Infants With Symptomatic Congenital Cytomegalovirus

Contact(s):

studyfinder@utsouthwestern.edu

Principal Investigator:

Gender: ALL

Age: 1 Day to 28 Days old

Phase: PHASE1

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT06118515

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Interventions: DRUG: Letermovir

Conditions: Congenital Cytomegalovirus Infection

Keywords: Congenital Cytomegalovirus Disease, Infants, Letermovir, Pharmacokinetic, Phase 1

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FrexalimAB in Preservation of Endogenous insULIN Secretion Compared to Placebo in adUlts and Adolescents on Top of inSulin Therapy (FABULINUS) (FABULINUS)

Contact(s):

studyfinder@utsouthwestern.edu

Principal Investigator:

Gender: ALL

Age: 12 Years to 35 Years old

Phase: PHASE2

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT06111586

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Inclusion Criteria:

* Participants who meet the criteria of T1D according to American Diabetes Association * Initiated exogenous insulin replacement therapy not longer than 90 days prior to screening visit at which random C-peptide will be assessed (V1). * Receiving at least one of the following T1D standard of care (SOC), insulin hormone replacement therapy * one or multiple daily injections (MDI) of basal insulin, prandial insulin and/or premixed insulin, or * continuous subcutaneous insulin infusion (CSII) * Participants must be positive for at least 1 of the following T1D autoantibodies confirmed by medical history and/or obtained at study screening: * Glutamic acid decarboxylase (GAD-65) * Insulinoma Antigen-2 (IA-2) * Zinc-transporter 8 (ZnT8) or * Insulin (if obtained not later than 10 days after exogenous insulin therapy initiation) * Have random C-peptide levels ≥ 0.2 nmol/L determined at screening. * Be vaccinated according to the local vaccination schedule. Any vaccinations should take place at least 28 days prior to randomization for non-live vaccines and at least 3 months prior to randomization for live vaccines. * Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies

Exclusion Criteria:

* Serious systemic viral, bacterial or fungal infection (eg, pneumonia, pyelonephritis), infection requiring hospitalization or IV antibiotics or significant chronic viral (including history of recurrent or active herpes zoster, acute or active cytomegalovirus (CMV), Epstein-Barr Virus (EBV) as determined at screening), bacterial, or fungal infection (eg, osteomyelitis) 30 days before and during screening. * Participants with a history of invasive opportunistic infections, such as, but not limited to histoplasmosis, listeriosis, coccidioidomycosis, candidiasis, pneumocystis jirovecii, and aspergillosis, regardless of resolution. * Evidence of active or latent tuberculosis (TB) as documented by medical history and examination, chest X-rays (posterior anterior and lateral), and/or TB testing. Blood testing (eg, QuantiFERON® TB Gold test) is strongly preferred; if not available, any local approved TB test is allowed. * Evidence of any clinically significant, severe or unstable, acute or chronically progressive, uncontrolled infection, medical or surgical condition (eg, but not limited to, cerebral, cardiac, pulmonary, renal, hepatic, gastrointestinal, neurologic, acquired or inherited bone/skeletal disorders including repeated bone fractures for unknown reason, juvenile osteoporosis, osteogenesis imperfecta, osteochondropathies, or any known immune deficiency), or any condition that may affect participant safety in the judgment of the Investigator (including vaccinations which are not updated based on local regulation). * History or current hypogammaglobulinemia. * History of a systemic hypersensitivity reaction or significant allergies, other than localized injection site reaction, to any humanized mAb. Clinically significant multiple or severe drug allergies, intolerance to topical corticosteroids, or severe post-treatment hypersensitivity reactions (including, but not limited to, erythema multiforme major, linear IgA dermatosis, toxic epidermal necrolysis, and exfoliative dermatitis). * Has other autoimmune diseases (eg, rheumatoid arthritis \[RA\], polyarticular juvenile idiopathic arthritis \[pJIA\], psoriatic arthritis \[PsA\], ankylosing spondylitis \[AS\], MS, SLE), except autoimmune thyroiditis with controlled function of thyroid gland and celiac disease (at discretion of investigator). * History, clinical evidence, suspicion or significant risk for thromboembolic events, as well as myocardial infarction, stroke, antiphospholipid syndrome, other prothrombotic disorders and/or participants requiring antithrombotic treatment. * Diabetes of forms other than autoimmune T1D that include but is not limited to genetic forms of diabetes, maturity-onset diabetes of the young (MODY), latent autoimmune diabetes of the adult (LADA), secondary to medications or surgery, type 2 diabetes by judgement of the investigator. * History of malignancy of any organ system, treated or untreated, within 5 years of screening, regardless of whether there is evidence of local recurrence or metastases. * Systemic corticosteroids (duration \> 7 days), adrenocorticotropic hormone 1 month prior to screening. * Any IV, IM or SC administered biologic treatments, \< 3 months or \< than 5 half-lives (whichever is longer), prior to randomization. * Any live (attenuated or viral-vector) vaccine (including but not limited to varicella zoster, oral polio, nasal influenza, rabies) within 3 months prior to randomization. * Any non-live (inactivated, mRNA, recombinant, conjugate, toxoid) vaccine administered less than 28 days prior to randomization. * Other medications not compatible or interfering with IMP at discretion of investigator. * Any immunosuppressive therapy within 12 weeks prior to randomization. * Course of Thymoglobulin®, teplizumab or other immunomodulatory treatments at any time. * Any glucagon-like peptide 1 (GLP-1) agonists and sodium-glucose co-transporter-2 and 1 (SGLT2/1) inhibitor and verapamil within 2 weeks prior to screening. * Abnormal laboratory test(s) at screening. The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Interventions: DRUG: Frexalimab, DRUG: Placebo, DRUG: Insulin

Conditions: Type 1 Diabetes Mellitus

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A Study Using Nivolumab, in Combination With Chemotherapy Drugs to Treat Nasopharyngeal Carcinoma (NPC)

Description:

This phase II trial tests effects of nivolumab in combination with chemotherapy drugs prior to radiation therapy patients with nasopharyngeal carcinoma (NPC). Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Chemotherapy drugs, such as gemcitabine and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high energy x-rays, particles, or radioactive seeds to kill cancer cells and shrink tumors. Researchers want to find out what effects, good and/or bad, adding nivolumab to chemotherapy has on patients with newly diagnosed NPC. In addition, they want to find out if children with NPC may be treated with less radiation therapy and whether this decreases the side effects of therapy.

Contact(s):

studyfinder@utsouthwestern.edu

Principal Investigator:

Gender: ALL

Age: to 21 Years old

Phase: PHASE2

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT06064097

Show full eligibility criteria

Inclusion Criteria:

* Patients must be ≤ 21 years of age at the time of study enrollment * Newly diagnosed American Joint Committee on Cancer (AJCC) stage II-IV nasopharyngeal carcinoma (NPC) * Patients must have had histologic verification of the malignancy at original diagnosis * Although submission of tumor tissue for the molecular characterization initiative is not required for eligibility, it is strongly recommended * Patients must have had histologic verification of the malignancy at original diagnosis * Although submission of tumor tissue for the molecular characterization initiative is not required for eligibility, it is strongly recommended * Patients must have a Lansky (for patients ≤ 16 years of age) or Karnofsky (for patients \> 16 years of age) performance status score of ≥ 60% * Peripheral absolute neutrophil count (ANC) ≥ 1000/uL (within 7 days prior to start of protocol therapy) * Platelet count ≥ 100,000/uL (transfusion independent) (within 7 days prior to start of protocol therapy) * Creatinine clearance or radioisotope glomerular filtration rate (GFR) ≥ 60 mL/min/1.73 m\^2 or (within 7 days prior to start of protocol therapy) * A serum creatinine based on age/gender (within 7 days prior to start of protocol therapy) Age: Maximum serum creatinine (mg/dL) 1 month to \< 6 months: 0.4 mg/dL (male); 0.4 mg/dL (female) 6 months to \< 1 year: 0.5 mg/dL (male); 0.5 mg/dL (female) 1 to \< 2 years: 0.6 mg/dL (male); 0.6 mg/dL (female) 2 to \< 6 years: 0.8 mg/dL (male); 0.8 mg/dL (female) 6 to \< 10 years 1 mg/dL (male); 1 mg/dL (female) 10 to \<13 years: 1.2 mg/dL (male); 1.2 mg/dL (female) 13 to \< 16 years: 1.5 mg/dL (male); 1.4 mg/dL (female) ≥ 16 years: 1.7 mg/dL (male); 1.4 mg/dL (female) * Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age, and (within 7 days prior to start of protocol therapy) * Serum glutamic-pyruvic transaminase (SGPT) (alanine aminotransferase \[ALT\]) ≤ 135 U/L\* (within 7 days prior to start of protocol therapy) * 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 radionuclide angiogram * No evidence of dyspnea at rest, no exercise intolerance, and a pulse oximetry \> 94% if there is clinical indication for determination * Human immunodeficiency virus (HIV)-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months and T-cell count above the lower limit of normal are eligible for this trial * For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated. 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

Exclusion Criteria:

* Patients who received prior radiotherapy to the head or neck * Patients who received prior chemotherapy or radiation for the treatment of any cancer in the last 3 years. These patients must also be in remission * Patients with a diagnosis of immunodeficiency * Patients with an active autoimmune disease that has required systemic treatment in the past 2 years (i.e., with use of disease-modifying agents, corticosteroids, or immunosuppressive agents). Replacement therapy (e.g., thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment. * Note: Patients with well-controlled asthma and no need for systemic steroids for the treatment of asthma in the last 12 months will not be excluded * Patients with a condition requiring systemic treatment with either corticosteroids (\> 0.25 mg/kg (10 mg) daily prednisone equivalent) within 14 days or other immunosuppressive medications within 30 days of enrollment. Inhaled or topical steroids, and adrenal replacement steroid doses \> 0.25 mg/kg (10 mg) daily prednisone equivalent, are permitted in the absence of active autoimmune disease * Patients with a history of (non-infectious) pneumonitis that required steroids or current pneumonitis * Patients with detectable viral load of human immunodeficiency virus (HIV), hepatitis B or hepatitis C, or active tuberculosis * Patients who have undergone solid organ or allogeneic hematopoietic transplant at any time * Due to risks of fetal and teratogenic adverse events as seen in animal studies, a negative pregnancy test must be obtained in females of childbearing potential, defined as females who are post-menarchal. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required * Females of childbearing potential that are sexually active must agree to either practice 2 medically accepted highly-effective methods of contraception at the same time or abstain from heterosexual intercourse from the time of signing the informed consent through 5 months after the last dose of nivolumab, 6 months after the last dose of gemcitabine, and 14 months after the last dose of cisplatin, whichever is longer * Males of childbearing potential that are sexually active must agree to either practice a medically accepted highly-effective methods of contraception or abstain from heterosexual intercourse from the time of signing the informed consent through 3 months after the last dose of gemcitabine, and 11 months after the last dose of cisplatin, whichever is longer * Lactating females are not eligible unless they have agreed not to breastfeed their infants starting with the first dose of study therapy through 5 months after the last dose of nivolumab * 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: Biopsy, PROCEDURE: Biospecimen Collection, PROCEDURE: Chest Radiography, DRUG: Cisplatin, PROCEDURE: Computed Tomography, PROCEDURE: Echocardiography, OTHER: Electronic Health Record Review, OTHER: Fluciclovine F18, DRUG: Gemcitabine, PROCEDURE: Magnetic Resonance Imaging, PROCEDURE: Multigated Acquisition Scan, BIOLOGICAL: Nivolumab, PROCEDURE: Positron Emission Tomography, OTHER: Quality-of-Life Assessment, OTHER: Questionnaire Administration, RADIATION: Radiation Therapy, PROCEDURE: X-Ray Imaging

Conditions: Stage II Nasopharyngeal Carcinoma AJCC v8, Stage III Nasopharyngeal Carcinoma AJCC v8, Stage IV Nasopharyngeal Carcinoma AJCC v8

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Liquid Biopsy in Ewing Sarcoma and Osteosarcoma As a Prognostic and Response Diagnostic: LEOPARD

Description:

This is a prospective multicenter biomarker study evaluating the prognostic impact of ctDNA detection at diagnosis in patients with Ewing sarcoma or osteosarcoma.

Contact(s):

studyfinder@utsouthwestern.edu

Principal Investigator:

Gender: ALL

Age: 12 Months to 50 Years old

Phase: NA

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT06068075

Show full eligibility criteria

Inclusion Criteria:

* For Part A, subjects must meet all of the following eligibility criteria. * Age: ≥ 12 months of age at time of study enrollment to 50 years of age * Diagnosis: Patients with histologic diagnosis (by institutional pathologist) of newly diagnosed, localized or regionally disseminated Ewing sarcoma or peripheral primitive neuroectodermal tumor (PNET) of bone or soft tissue or; Patients with histologic diagnosis (by institutional pathologist) of newly diagnosed, non-pelvic, localized or regionally disseminated high-grade osteosarcoma. NOTE: Staging will be assessed according to standard of care at the treating center. * Prior Therapy: * Patients should have only previously had a biopsy, and not had prior attempt at tumor resection. * Not yet started chemotherapy or radiation therapy OR patient has started chemotherapy or radiation therapy, but an appropriate pre-treatment baseline sample was collected and processed for ctDNA under a local banking study in DFCI Pediatrics and is available to use for this study. * Planned to receive chemotherapy as follows: -- VDC/IE as per COG protocols AEWS0031, AEWS1031 or AEWS1221 (for patients with Ewing sarcoma or PNET); or MAP as per COG protocol AOST0331 (for patients with osteosarcoma). * For Part B subjects must meet all of the following eligibility criteria. * Age: ≥ 12 months of age at time of study enrollment * Diagnosis: Patients with histologic diagnosis (by institutional pathologist) of newly diagnosed Ewing sarcoma or peripheral primitive neuroectodermal tumor (PNET) of bone or soft tissue * Prior Therapy: * Patients should have had only frontline therapy as per institutional standard, and maintenance therapy if given (no relapse therapy). * If frontline systemic therapy already completed (not including maintenance or metastatic site radiation), therapy completed within 6 months of enrollment to Part B. * Subjects must have a willing physician provider supporting their participation in Part B. * For Part B, providers are eligible to receive the provider survey if they are listed as the primary provider for the patient at the study site.

Exclusion Criteria:

* For Part A, subjects must not meet any of the following exclusion criteria. * Patients with distant metastatic disease. * Patients with known Ewing-like sarcoma (e.g., BCOR-CCNB3 or CIC-DUX4 translocated small round cell sarcomas) are not eligible. * Patients who are enrolled with an initial diagnosis of Ewing sarcoma and subsequently found to have Ewing-like sarcoma will be replaced. Samples obtained prior to removal from study will be analyzed and reported descriptively. Patients with Ewing-like tumors may continue to provide samples and clinical data until they meet off-study criteria per protocol. * Patients weighing \< 5 kg at time of diagnosis * Patients with a second malignant neoplasm * Patients without detectable tumor at the time of study enrollment (ie, complete tumor resection prior to study enrollment) * Patients already receiving tumor-directed therapy at the time of study enrollment except when a pre-treatment baseline sample has already been obtained under a local banking study in DFCI Pediatrics that would be eligible for analysis under this study. * Patients with osteosarcoma with a pelvic primary tumor site Pregnancy * For Part B, subjects must not meet any of the following exclusion criteria. * Patients with known Ewing-like sarcoma (e.g., BCOR-CCNB3 or CIC-DUX4 translocated small round cell sarcomas) are not eligible. * Samples obtained prior to removal from study will be analyzed and reported descriptively. Patients with Ewing-like tumors may continue to provide samples and clinical data until they meet off-study criteria per protocol * Patients weighing \< 5 kg at time of enrollment * Patients diagnosed with relapsed disease and/or having started therapy directed at disease relapse * Pregnancy * Resides outside of the United States * For Part B, providers at non-study centers will not be eligible to receive the provider survey.

Interventions: OTHER: FoundationOne Liquid CDx

Conditions: Ewing Sarcoma, Ewing Sarcoma of Bone, Ewing Sarcoma of Soft Tissue, Peripheral Primitive Neuroectodermal Tumor, Peripheral Primitive Neuroectodermal Tumor of Bone, Peripheral Primitive Neuroectodermal Tumor of Soft Tissue, High-grade Osteosarcoma

Keywords: Ewing Sarcoma, Ewing Sarcoma of Bone, Ewing Sarcoma of Soft Tissue, Peripheral Primitive Neuroectodermal Tumor, Peripheral Primitive Neuroectodermal Tumor of Bone, Peripheral Primitive Neuroectodermal Tumor of Soft Tissue, High-grade osteosarcoma

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Massive Transfusion in Children-2: A Trial Examining Life Threatening Hemorrhage in Children (MATIC-2)

Description:

The MATIC-2 is a multicenter clinical trial enrolling children who are less than 18 years of age with hemorrhagic shock potentially needing significant blood transfusion. The primary objective of the clinical trial is to determine the effectiveness of Low Titer Group O Whole Blood (LTOWB) compared to component therapy (CT), and Tranexamic Acid (TXA) compared to placebo in decreasing 24-hour all-cause mortality in children with traumatic life threatening hemorrhage.

Contact(s):

studyfinder@utsouthwestern.edu

Principal Investigator:

Gender: ALL

Age: to 17 Years old

Phase: PHASE3

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT06070350

Show full eligibility criteria

Interventions: BIOLOGICAL: Low Titer Group O Whole Blood (LTOWB), DRUG: Placebo, DRUG: Tranexamic Acid (TXA), BIOLOGICAL: Component Therapy (CT)

Conditions: Hemorrhagic Shock, Trauma Injury

Keywords: Children, Pediatrics, Transfusion

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Studying the Health of Asians to Advance Knowledge, Treatments, and Interventions for Depression (SHAKTI)

Description:

SHAKTI (from the Sanskrit word for "power") is a 5-year natural history, longitudinal, prospective study of a cohort of 6,000 participants that will help uncover the socio-demographic, lifestyle, clinical, psychological, and neurobiological factors that contribute to antidepressant treatment response (remission, recurrence, relapse and individual outcomes in depressive disorders) and resilience. As this is an exploratory study, we will assess a comprehensive panel of carefully selected participant specific parameters - socio-demographic (age, sex, gender, race, ethnicity, economic); life habits (physical activity, substance use); clinical (medical history, anxious depression, early life trauma), biological (biomarkers in blood, saliva, urine, stool), behavioral (cognitive, emotional), neurophysiological (EEG), and neuroimaging (magnetic resonance imaging; MRI) with the goal of developing the most robust predictive models of depression treatment response and of outcomes.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, Afrida.Khurshid@UTSouthwestern.edu

Principal Investigator: Madhukar Trivedi

Gender: ALL

Age: 10 Years and over

Phase:

Healthy Volunteers:

This study is also accepting healthy volunteers

System ID: NCT06002204

IRB Number: STU-2023-0616

Show full eligibility criteria

Interventions: OTHER: Observational Study

Conditions: Depression, Treatment Resistant Depression, Mood Disorders, Bipolar Disorder, Mental Disorders, Bipolar and Related Disorders

Keywords: Depression, Resilience, Asian & Pacific Islanders, Mood Disorders, Bipolar Disorder, Behavioral Symptoms, Observational Study

Sites: UT Southwestern

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A Master Protocol (LY900023) That Includes Several Clinical Trials of Drugs for Children and Young Adults With Cancer (CAMPFIRE)

Description:

The main purpose of the master is to help the research sites and sponsor carry out several clinical trials more efficiently by providing a common research protocol. Individual clinical trials under this master protocol define drug/disease-specific research goals and activities to test them. New studies will be added as new drugs emerge against different cancers. Participation in the trial will depend on how long the benefit lasts.

Contact(s):

studyfinder@utsouthwestern.edu

Principal Investigator:

Gender: ALL

Age: 1 Year to 39 Years old

Phase: PHASE2

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05999994

Show full eligibility criteria

Interventions: DRUG: Ramucirumab, DRUG: Cyclophosphamide, DRUG: Vinorelbine, DRUG: Gemcitabine, DRUG: Docetaxel, DRUG: Abemaciclib, DRUG: Irinotecan, DRUG: Temozolomide

Conditions: Neoplasms, Child, Adolescent

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NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)

Description:

This is a Phase 2, open-label, multi-center, 2-part study of NS-089/NCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to <15 years with DMD due to mutations amenable to exon 44 skipping. Participants will receive a selected dose of NS-089/NCNP-02 administered once weekly. The study consists of 2 parts: Part 1 and Part 2. Six participants (Cohort 1) will participate in both Part 1 and Part 2, and 14 participants (Cohort 2) will be added for Part 2.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, Deniece.Winston@UTSouthwestern.edu

Principal Investigator: Kaitlin Batley

Gender: MALE

Age: 4 Years to 14 Years old

Phase: PHASE2

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05996003

IRB Number: STU-2022-0205

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Interventions: DRUG: NS-089/NCNP-02

Conditions: Duchenne Muscular Dystrophy

Sites: Children’s Health

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A Study to Evaluate Long-term Safety of Ecopipam Tablets in Children, Adolescents and Adults With Tourette's Disorder

Description:

The primary objective of this study is to evaluate the long-term safety and tolerability of ecopipam tablets in children (greater than or equal to \[\>=\] 6 and less than \[\<\] 12 years of age), adolescents (\>=12 and \<18 years of age), and adults (\>=18 years of age) with Tourette's Syndrome (TS).

Contact(s):

Lauren Sanchez Dengle, MD lauren.dengle@utsouthwestern.edu

Principal Investigator:

Gender: ALL

Age: 6 Years and over

Phase: PHASE3

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT06021522

Show full eligibility criteria

Inclusion Criteria:

* \>=6 to \>=18 years of age. * Participants enrolling from the study EBS-101-TD-301; completed all visits through Week 24 and days 7 and 14 safety follow-up, met relapse criteria during the double-blind randomized (R/WD) period after completing the 301 end of trial (ET) visit, the 7 day and 14 day safety follow up visits, but not before 24 weeks following the 301 baseline visit or participants who met relapse criteria will be eligible after completing early termination visit, Day 7 and Day 14 follow up visits. * Participants who completed the studies EBS-101-OL-001 or PSY302A. * The enrolling participant must have had clinical benefit from ecopipam and would benefit from continued participation. * Effective contraception during the study and 30 days after last study dose for sexually active participants * \<18 years of age participants parent/legal guardian must sign a written informed consent and participant must sign a written informed assent. * Participant must have TD based on Diagnostic and Statistical Manual for Mental Disorders - 5th Edition (DSM-5-TR diagnostic criteria) for TD. * TD diagnosis and both motor and vocal tics that cause impairment with normal routines

Exclusion Criteria:

* The participants who discontinued the studies PSY-302A, EBS-101-OL-001 or EBS-101-TD-301 due to reasons such as either lost to follow up, withdrawn consent, non-compliant or withdrawn by the discretion of either the site investigator or the sponsor. * Participants with ongoing or past history of neurological condition (example \[e.g.\], Huntington's disease, Parkinson's disease, Wilson's disease, stroke, Restless Legs Syndrome). * Any unstable mood disorder (DSM-5-TR criteria), mental illness or clinically significant lab abnormalities, moderate to severe renal or hepatic impairment, a PHQ-9 score \>=10 at screening and history of neuroleptic malignant syndrome at the time of screening or baseline. * Participants who completed the studies EBS-101-OL-001 or PSY-302A and who had previous exposure to ecopipam and oral neuroleptics within 4 weeks and depot neuroleptics within 3 months prior to screening, 6 months prior to Baseline. * Participants receiving any other medication to treat motor or vocal tics and anti-depressant or anti-anxiety medications. * Risk of suicide as per PI judgement * Pregnant or lactating women * Certain medications that would have unfavorable drug interactions with ecopipam, e.g., digoxin, fluoxetine, valproic acid, bupropion. * Current or recent (past 3 months) DSM-5-TR substance use disorder (with the exception of nicotine). * Recent behavioral therapy * Positive urine drug screen for cocaine, amphetamine, benzodiazepines, barbiturates, phencyclidine (PCP) or opiates at Baseline, except those receiving stable, prescribed treatment for attention deficit/hyperactivity disorder (ADHD) * Lifetime history of bipolar disorder type I or II, dementia, schizophrenia, or any other psychotic disorder. * Unable to swallow tablets. * Known hypersensitivity to any of ecopipam's excipients. * History of seizures (excluding febrile seizures that occurred \>2 years prior to Baseline). * Myocardial infarction within 6 months from Screening.

Interventions: DRUG: Ecopipam

Conditions: Tourette Syndrome

Keywords: Ecopipam, Neurodevelopmental Disorders, Mental Disorders, Central Nervous System Depressants, Neurodegenerative Diseases, Movement Disorders, Tic Disorders, Nervous System Diseases, Central Nervous System Diseases, Brain Diseases

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Nuwiq for Perioperative Management Of Patients With Haemophilia A on Emicizumab Regular Prophylaxis Study (NuPOWER)

Contact(s):

studyfinder@utsouthwestern.edu

Principal Investigator:

Gender: MALE

Age: 12 Years to old

Phase: PHASE4

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05935358

Show full eligibility criteria

Interventions: DRUG: Nuwiq

Conditions: Severe Hemophilia A

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A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A

Description:

This is a prospective, observational, multi-center longitudinal cohort study to describe the real-world effectiveness, safety and treatment usage of efanesoctocog alfa in patients with hemophilia A treated per standard of care in the US and Japan. Patients will be enrolled in the study after the introduction of efanesoctocog alfa in the hemophilia treatment landscape in each study country. Decision to initiate treatment with commercially available efanesoctocog alfa will be made by the treating physician independently from the decision to include patients in the study. No study medication is provided. The data related to efanesoctocog alfa effectiveness, safety and usage will be collected prospectively during routine visits (expected annual/semi-annual visits) for up to 5 years following enrollment /treatment initiation.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, lindsey.hartland@childrens.com

Principal Investigator: Jessica Garcia

Gender: ALL

Age: Not specified

Phase:

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05911763

IRB Number: STU-2023-0545

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Interventions: DRUG: Efanesoctocog Alfa BIVV001

Conditions: Hemophilia A

Sites: Children’s Health

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Optimization of Saturation Targets And Resuscitation Trial (OptiSTART) (OptiSTART)

Description:

This study is designed to answer one of the fundamental gaps in knowledge in the resuscitation of preterm infants at birth: What is the optimal target oxygen saturation (SpO2) range that increases survival without long-term morbidities? Oxygen (O2) is routinely used for the stabilization of preterm infants in the delivery room (DR), but its use is linked with mortality and several morbidities including bronchopulmonary dysplasia (BPD). To balance the need to give sufficient O2 to correct hypoxia and avoid excess O2, the neonatal resuscitation program (NRP) recommends initiating preterm resuscitation with low (≤ 30%) inspired O2 concentration (FiO2) and subsequent titration to achieve a specified target SpO2 range. These SpO2 targets are based on approximated 50th percentile SpO2 (Sat50) observed in healthy term infants. However, the optimal SpO2 targets remain undefined in the preterm infants. Recent data suggest that the current SpO2 targets (Sat50) may be too low. The investigators plan to conduct a multicenter RCT of Sat75 versus Sat50 powered for survival without BPD. The investigators will randomize 700 infants, 23 0/7- 30 6/7 weeks' GA, to 75th percentile SpO2 goals (Sat75, Intervention) or 50th percentile SpO2 goals (Sat50, control). Except for the SpO2 targets, all resuscitations will follow NRP guidelines including an initial FiO2 of 0.3. In Aim 1, the investigators will determine whether targeting Sat75 compared to Sat50 increases survival without lung disease (BPD). In addition, the investigators will compare the rates of other major morbidities such as IVH. In Aim 2, the investigators will determine whether targeting Sat75 compared to Sat50 increases survival without neurodevelopmental impairment at 2 years of age. In Aim 3, the investigators will determine whether targeting Sat75 compared to Sat50 decreases oxidative stress.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, shelby.unger@UTSouthwestern.edu

Principal Investigator: Vishal Kapadia

Gender: All

Age: 0 Minutes to 10 Minutes old

Phase: N/A

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05849077

IRB Number: STU-2022-0441

Show full eligibility criteria

Interventions: Other: Sat75, Other: Sat50

Conditions: Premature Infants, Bronchopulmonary Dysplasia, Intraventricular Hemorrhage, Neurodevelopmental Outcomes

Keywords: neonatal resuscitation, oxygen

Sites: Parkland Health & Hospital System

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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

Show full eligibility criteria

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)

Contact(s):

studyfinder@utsouthwestern.edu

Principal Investigator:

Gender: ALL

Age: 3 Months to 24 Months old

Phase: PHASE4

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05861986

Show full eligibility criteria

Interventions: DRUG: risdiplam

Conditions: Muscular Atrophy, Spinal

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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

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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/gender 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. Participants (male and female) who are sexually active must use two forms of an acceptable method of birth control (for men, one form must be a barrier method) from start of therapy through 180 days following last dose of tovorafenib (DAY101)

Interventions: PROCEDURE: Biospecimen Collection, PROCEDURE: Bone Marrow Aspiration, PROCEDURE: Bone Marrow Biopsy, PROCEDURE: Computed Tomography, PROCEDURE: Echocardiography, 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

Sites: Children’s Health

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Long Term Efficacy and Safety of Orlistat for Type 1 Hyperlipoproteinemia

Description:

Type I hyperlipoproteinemia (T1HLP, also known as familial chylomicronemia syndrome or FCS) is a rare diseasewhere the blood triglycerides (fats) are very high. It is caused by lack of certain enzymes and proteins in the bodythat are important in disposing circulating fats from blood. Treatment of T1HLP patients who have very high levels of blood fats (≥ 1,000 mg/dL) is challenging as conventional triglyceride-lowering medications, such as fibrates and fishoil, are ineffective. The purpose of this trial is to study the long-term efficacy and safety of orlistat for reducing blood triglyceride levels in patients with T1HLP.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, CHANDNA.VASANDANI@UTSouthwestern.edu

Principal Investigator: Abhimanyu Garg

Gender: All

Age: 8 Years to 70 Years old

Phase: Phase 2

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05816343

IRB Number: STU-2019-0776

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Interventions: Drug: Orlistat, Drug: Placebo

Conditions: Type 1 Hyperlipoprotenemia

Sites: UT Southwestern; Parkland Health & Hospital System

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A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace) (PALace)

Description:

This is a 10-year multi-center, global, observational study to further characterize the safety profile of pegvaliase, including hypersensitivity reactions, long-term safety and tolerability, and the effectiveness of the additional risk minimization measures (aRMMs) (European Union (EU) only) in subjects receiving pegvaliase for the treatment of PKU. Subjects for whom a clinical decision has been made that they will receive pegvaliase to treat their PKU within 30 days following the date of enrollment (incident-users) or have previously started treatment with pegvaliase at the date of enrollment (prevalent-users) are eligible for participation in this study.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, Juana.Luevano@UTSouthwestern.edu

Principal Investigator: Markey McNutt

Gender: All

Age: Not specified

Phase:

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05813678

IRB Number: STU-2023-0263

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Interventions: Drug: Pegvaliase

Conditions: Phenylketonuria (PKU), Other Endocrine System

Keywords: Observational, Safety Study, Pegvaliase, Palynziq, PKU, Phenylketonuria, Phase 4

Sites: UT Southwestern; Children’s Health

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Safety and Efficacy of Radio Frequency for the Treatment of Mild to Severe Inflammatory Acne

Description:

The aim of this trial is to evaluate the safety and efficacy of the InMode RF Pro System with the Morpheus8 face tip (24 pins) applicator for the treatment of mild, moderate and severe, facial acne vulgaris

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, JENNIFER.BARILLAS@UTSouthwestern.edu

Principal Investigator: Jeffrey Kenkel

Gender: ALL

Age: 16 Years and over

Phase: NA

Healthy Volunteers:

This study is also accepting healthy volunteers

System ID: NCT05830968

IRB Number: STU-2023-0661

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Inclusion Criteria:

* Subject is \>16 years of age * General good health confirmed by medical history and examination of the treated area. * Subjects with mild to severe Acne Vulgaris, defined as a baseline IGA (Investigator's Global Assessment) score of 2, 3 or 4 and 10-100 inflammatory lesions (papules or pustules). * The patients should be willing to comply with the study procedure and schedule, including the follow up visits, and will refrain from using any other acne treatment methods during the entire study period. * Willing to avoid sun/UV exposure for duration of the study unless using sunscreen. * Willing to refrain from starting or changing hormonal contraception for duration of study. * Subject understands and is willing to sign the informed consent to participate in the study. Parental (or other) guardians must provide consent for minors under the age of 18.

Exclusion Criteria:

- Pacemaker or internal defibrillator, or any other active electrical implant anywhere in the body. * Patients who are under pharmacological anti-acne therapy (isotretinoin or antibiotics) for the last 6 months. * Use of botulinum toxin within prior 1 month. * Permanent implant in the treated area such as metal plates and screws, silicone implants or an injected chemical substance * Current or history of cancer, or premalignant condition in the treatment area. * Severe concurrent conditions, such as cardiac disorders, epilepsy, uncontrolled hypertension, and liver or kidney diseases. * Subject who are pregnant or nursing. * Started or changed hormonal contraceptive within prior month of study. * Subject is unwilling or unlikely to refrain from high UV exposure to face. * Impaired immune system due to immunosuppressive diseases such as AIDS and HIV, or use of immunosuppressive medications. * Patients with history of diseases stimulated by heat, such as recurrent Herpes Simplex in the treatment area * Poorly controlled endocrine disorders, such as diabetes or thyroid dysfunction. * Any active condition in the treatment area, such as sores, psoriasis, eczema, and rash. * History of skin disorders, keloids, abnormal wound healing, as well as very dry and fragile skin. * History of bleeding coagulopathies or use of anticoagulants in the last 10 days. * Any surgery in treated area within 3 months prior to treatment. * Subject received other treatments such as light, CO2 laser or RF in the treatment area within 6 months of study start date. * Simultaneous participation in another investigator drug or device study or completion of the follow-up phase for the primary endpoint of any previous study less than 30 days prior to the first evaluation in this study. * Subject that has any condition that, at the investigator's discretion, renders the subject unsuitable for participation in this clinical research study.

Interventions: DEVICE: Morpheus8 Applicator Radiofrequency device

Conditions: Inflammatory Acne Vulgaris, Other Skin

Sites: UT Southwestern

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Natural History Study of Participants With Sanfilippo Syndrome Type IIIC (MPSIIIC)

Description:

This study is planned to document, through retrospective and prospective data collection, syndrome progression in children and young adults with MPS IIIC.

Contact(s):

studyfinder@utsouthwestern.edu

Principal Investigator:

Gender: ALL

Age: 12 Months and over

Phase:

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05825131

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Conditions: Sanfilippo Syndrome Type C

Keywords: Sanfilippo syndrome type C, Mucopolysaccharidosis type III C, MPS IIIC

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Safety and Efficacy Study of Viaskin Peanut in Peanut-allergic Children 4-7 Years of Age (VITESSE)

Description:

The primary purpose of this study is to assess the efficacy and safety of daily DBV712 250 micrograms (mcg) to induce desensitization to peanut in peanut-allergic children 4-7 years of age over a 12-month treatment period.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, Priscilla.Arancivia@UTSouthwestern.edu

Principal Investigator: Christopher Parrish

Gender: All

Age: 4 Years to 7 Years old

Phase: Phase 3

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05741476

IRB Number: STU-2023-0051

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Interventions: Drug: DBV712, Other: Placebo

Conditions: Allergy, Peanut, Other

Keywords: Peanut Hypersensitivity, Epicutaneous Immunotherapy (EPIT), Epicutaneous, Immunotherapy, Viaskin, Nut and Peanut Hypersensitivity, Food Hypersensitivity, Peanut Allergy, Food Allergy, Nut and Peanut Allergy

Sites: Children’s Health

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Janus Kinase (JAK) Inhibitors to Preserve C-Peptide Production in New Onset Type 1 Diabetes (T1D)

Description:

A multi-center, placebo-controlled, double blind, 1:1:1 randomized control clinical trial testing two different JAK Inhibitors abrocitnib, ritlecitinib, and placebo in subjects with recent onset Stage 3 Type 1 Diabetes within 100 days of diagnosis.

Contact(s):

Call 214-648-5005
studyfinder@utsouthwestern.edu, Michelle.Murphy@UTSouthwestern.edu

Principal Investigator: Perrin White

Gender: ALL

Age: 12 Years to 35 Years old

Phase: PHASE2

Healthy Volunteers:

This study is NOT accepting healthy volunteers

System ID: NCT05743244

IRB Number: STU-2023-1068

Show full eligibility criteria

Inclusion Criteria:

• Provide informed consent or assent as appropriate and, if \< 18 years of age have a parent or legal guardian provide informed consent
• Age 12-35 years (both inclusive) at the time of signing informed consent and assent
• Diagnosis of T1D within 100 days of the baseline visit (V0).
• Positive for at least one islet cell autoantibody; Glutamate decarboxylase (GAD)65A, mIAA (if obtained within 10 days of the onset of insulin therapy), IA-2A, ICA, or ZnT8A
• Stimulated C-peptide of ≥0.2 pmol/mL measured during mixed-meal tolerance test (MMTT) conducted at least 21 days from diagnosis of diabetes
• HbA1c ≤ 10 %
• Body weight ≥ 35kg at screening
• Willing to comply with intensive diabetes management and wear a Continuous Glucose Monitoring Device (CGM)
• Participants who are Cytomegalovirus (CMV) and/or Epstein-Barr virus (EBV) seronegative at screening must be CMV and/or EBV Polymerase chain reaction (PCR) negative within 30 days of randomization and may not have had signs or symptoms of a CMV and/or EBV-compatible illness lasting longer than 7 days within 30 days of the baseline visit (V0).
• Participants who are CMV and/or EBV seropositive at screening must be CMV PCR negative and/or EBV PCR \<2,000 IU/mL and must have no signs or symptoms of acute infection at the time of the baseline visit (V0).
• Be up to date on recommended vaccinations based on age of participants\*
• Participants are required to receive killed influenza vaccination at least 2 weeks prior to the baseline visit (V0) when vaccine for the current or upcoming flu season is available. Enrollment must be delayed at least 4 weeks from administration of a killed vaccine other than influenza and COVID-19 and 6 weeks from a live vaccination. Live vaccinations and non-live vaccinations (other than influzena and COVID-19) should not be given while on study drug and be postponed at least 3 months after the last dose of study drug.
• If participant is female with reproductive potential, she must have a negative pregnancy test at screening and be willing to avoid pregnancy using a highly-effective contraceptive method for the duration of the study
• Males of reproductive age must use a highly-effective contraceptive method during the treatment phase and for 3 months following last dose of study drug * For COVID-19 vaccination, all participants will be strongly encouraged to be up-to-date with COVID-19 vaccine (s) as indicated by country-specific guidelines at least 2 weeks prior to the baseline visit (V0).

Exclusion Criteria:

• Current or ongoing use of non-insulin pharmaceuticals or medication that affect glycemic control or glucose homeostasis within 7 days prior to screening or any prohibited concomitant medication listed in section 4.8
• Untreated hypothyroidism or active Graves' disease
• Concurrent treatment with other immunosuppressive agents (including biologics or steroids), other than inhaled or topical glucocorticoids
• Active acute or chronic infection requiring treatment with oral antibiotics, antivirals, antiparasitics, antiprotozoals, or antifungals within 1 month prior to Day 0 or superficial skin infection within 1 week prior to Day 0
• Active acute or chronic infection requiring treatment with intravenous therapy (IV) within a minimum 1 month prior to Day 0 a. Specific cases should be reviewed by Infectious Disease Committee prior to enrollment
• Have active signs or symptoms of acute infection at the time of the baseline visit (V0).
• Significant trauma or major surgery within 1 month of signing informed consent.
• Considered in imminent need for surgery or with elective surgery scheduled to occur during the study
• History of disseminated herpes zoster or disseminated herpes simplex or a recurrent (more than one episode of) localized, dermatomal herpes zoster
• Have evidence of prior or current tuberculosis infection as assessed by Purified Protein Derivative (PPD), interferon gamma release assay (IGRA) or by history
• Have evidence of current or past HIV or Hepatitis B infection
• Have evidence of active Hepatitis C infection
• Have current, confirmed COVID-19 infection
• Current or history of Deep vein thrombosis (DVT), Pulmonary embolism (PE), or other thromboembolic events or history of inherited coagulopathies
• First degree relative with a history of unprovoked venous thromboembolism (i.e. without known underlying cause such as trauma, surgery, immobilization, prolonged travel, pregnancy, hormone use, or plaster cast), which suggests that a participant may be at increased risk of inherited coagulation disorder
• Any present malignancies or history of malignancy, other than a successfully treated nonmelanoma skin cancer
• History of any lymphoproliferative disorder such as EBV-related lymphoproliferative disorder, history of lymphoma, history of leukemia, or signs and symptoms suggestive of current lymphatic or lymphoid disease
• Known or suspected polymorphism in the Cytochrome P450 2C19 (CYP2C19 gene, resulting in classification as a poor CYP2C19 metabolizer).
• Have renal impairment (eGFR\< 60 mL/min)
• Currently on anti-platelet therapies, excluding low dose aspirin
• One or more screening laboratory values as stated
• Neutrophils \< 1,500 /μL
• Lymphocytes \< 800 /μL
• Platelets \< 150,000 / μL
• Hemoglobin \< 6.2 mmol/L (10.0 g/dL)
• Potassium \> 5.5 mmol/L or \<3.0 mmol/L
• Sodium \> 150mmol/L or \< 130mmol/L
• AST or ALT ≥ 2.5 times the upper limit of normal
• Bilirubin ≥ 1.5 times upper limit of normal unless diagnosed with Gilbert's syndrome
• LDL \>160 mg/dL
• Vaccination with a live virus within the last 6 weeks and killed vaccine within 4 weeks (except 2 weeks for flu vaccine and COVID vaccine)
• Be currently pregnant or lactating or anticipate becoming pregnant during the study
• Male participants able to father children and female participants of childbearing potential who are unwilling or unable to use 2 effective methods (at least 1 highly effective method) of contraception, including abstinence, as outlined in this protocol for the duration of the study and for at least 3 months after the last dose of investigational product
• Be currently participating in another T1D treatment study
• Have hearing loss with progression over the previous 5 years, or sudden hearing loss, or middle or inner ear disease such as otitis media, cholesteatoma, Meniere's disease, labyrinthitis, or other auditory condition that is considered acute, fluctuating, or progressive
• Acute coronary syndrome (e.g., myocardial infarction, unstable angina pectoris) and any history of cerebrovascular disease within 24 weeks before screening; Heart failure NYHA (New York Heart Association) III, NYHA IV
• ANY of the following conditions at screening: a. Screening 12-lead electrocardiogram (ECG) that demonstrates: i. Clinically significant abnormalities requiring treatment (eg, acute myocardial infarction, serious tachy- or brady-arrhythmias) or indicating serious underlying heart disease (eg, cardiomyopathy, Wolff-Parkinson- White syndrome); ii. Confirmed QT corrected using Fridericia's correction factor (QTcF) prolongation (\>450 milliseconds). b. Long QT Syndrome, a family history of Long QT Syndrome, or a history of Torsades de Pointes (TdP).
• History of chronic alcohol abuse or intravenous drug abuse or other illicit drug abuse within 2 years prior to screening
• Current or past use of tobacco or nicotine containing products more than the equivalent of 5 cigarettes per day
• Participant is the investigator or any sub-investigator, research assistant, pharmacist, study coordinator, other staff or relative thereof directly involved in the conduct of the trial
• Have any complicating medical issues or abnormal clinical laboratory results that may interfere with study conduct, or cause increased risk
• Any condition that in the investigator's opinion may adversely affect study participation or may compromise the study results

Interventions: DRUG: Abrocitinib 200 MG Oral Tablet, DRUG: Ritlecitinib, DRUG: Placebo

Conditions: Diabetes Mellitus, Type 1, Pancreas

Keywords: TrialNet, T1D

Sites: UT Southwestern; Children’s Health

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