Here are the studies that match your search criteria. If you are interested in participating, please reach out to the contact listed for the study. If no contact is listed, contact us and we'll help you find the right person.
DExterous Hand Control Through Fascicular Targeting (DEFT) - (Human Subjects)
Our goal is to temporarily implant the following groups for 180 +/- 30 days:
1. Five human partial hand amputees (amputated at the level of the hand) with 2 FAST-LIFE
electrodes, one inserted into the motor fascicle of the ulnar nerve and the other into
the sensory fascicle.
2. Five human hand and forearm amputees (amputated at the level of the forearm) with 2
FAST-LIFE electrodes in the ulnar nerve (one in the motor fascicle, one in the sensory
fascicle) and 2-5 FAST-LIFE electrodes in the median nerve (one in the motor fascicle,
one to four in the remaining sensory fascicles).
Call 214-648-5005 studyfinder@utsouthwestern.edu
Jonathan Cheng
98715
All
18 Years to 95 Years old
N/A
This study is NOT accepting healthy volunteers
NCT02994160
STU 092014-061
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Criteria for Inclusion of Subjects:
Hand and forearm amputees:
1. Male or female, age 18 and older, of any race or ethnicity
2. Able and willing to sign Consent
3. Able and willing to participate in all study activities including implantation,
testing and explantation of the study device.
4. Able to communicate effectively in English without an interpreter
After preliminary screening subjects will be assessed for the following inclusion criteria:
1. Patient has an existing myoelectric hand prosthesis and demonstrates proficiency
during daily use
2. Overall and phantom pain are well-controlled and not incapacitating
Criteria for Exclusion of Subjects:
1. If MR neurogram and EMG/NCS study show nerve or muscle dysfunction/injury at a higher
level than anticipated based on the appearance of the physical amputation stump, the
subject may be excluded from the study due to adverse neuromuscular anatomy which
would preclude use of the proposed experimental electrode implants. The radiographs
will be used to confirm suitability of the amputation stump configuration. If the bony
anatomy of the amputation stump is found to be unsuitable, the patient may be excluded
from the study.
2. Subjects who have a history of cardiac arrhythmia will be excluded from the study.
Other: FastLIFE electrode
Amputation, Traumatic, Hand, Brain and Nervous System
peripheral nerve, intraneural electrode, hand amputation, forearm amputation
Phase 1 Study of MM-398 Plus Cyclophosphamide in Pediatric Solid Tumors
This is a Phase 1 study of the combination of two drugs: MM-398 and Cyclophosphamide. The
goal is to find the highest dose of MM-398 that can be given safely when it is used together
with the chemotherapy drug Cyclophosphamide.
• Histologically or cytologically-confirmed Ewing sarcoma, rhabdomyosarcoma,
neuroblastoma, or osteosarcoma
• Disease progression after prior therapy in locally advanced or metastatic setting
• Measurable or evaluable disease based on the Response Evaluation Criteria in Solid
Tumors (RECIST v1.1) criteria
• Age 12 months to <21 years
• Adequate bone marrow reserves, hepatic function, and renal function
• Recovered from effects of any prior surgery or cancer therapy
• Patients 18 years or older will provide written consent. A parent or legal guardian of
a patient <18 years of age will provide informed consent and patients 11 to 18 years
of age will provide written assent or as per participating institutional policy.
Exclusion Criteria:
• Clinically significant gastrointestinal disorders
• NYHA Class III or IV congestive heart failure, ventricular arrhythmias or uncontrolled
blood pressure
• Active infection or unexplained fever
• Known hypersensitivity to any of the components of MM-398 or other liposomal products
• Recent Investigational therapy
• Pregnant or breast feeding; females of child-bearing potential must test negative for
pregnancy at the time of enrollment
Drug: MM-398 (Irinotecan Sucrosofate Liposome Injection) plus cyclophosphamide
Neuroblastoma, Sarcoma, Ewing Sarcoma, Mycosis Fungoides, Rhabdomyosarcoma, Osteosarcoma, Recurrent or Refractory Solid Tumors, Brain and Nervous System, Eye and Orbit, Anus, Bones and Joints, Breast - Female, Breast - Male, Cervix, Colon, Corpus Uteri, Ear, Esophagus, Gall Bladder, Head and Neck, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Melanoma, skin, Nose, Other Digestive Organ, Other Endocrine System, Other Female Genital, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Ovary, Pancreas, Prostate, Rectum, Stomach, Throat, Thyroid, Urinary Bladder, Uterine (Endometrial), Vulva, Hodgkins Lymphoma, Kaposis sarcoma, Other Hematopoietic, Small Intestine, Soft Tissue
Study of SRP-4045 and SRP-4053 in DMD Patients (ESSENCE)
The main objective of this study is to evaluate the efficacy of SRP-4045 and SRP-4053
compared to placebo in Duchenne muscular dystrophy (DMD) patients with out-of-frame deletion
mutations amenable to skipping exon 45 and exon 53, respectively.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Susan Iannaccone
13463
Male
7 Years to 13 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT02500381
STU 082015-050
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Inclusion Criteria:
• Genotypically confirmed DMD, with genetic deletion amenable to exon 45 or exon 53
skipping
• Stable dose of oral corticosteroids for at least 24 weeks
• Intact right and left biceps or 2 alternative upper muscle groups
• Mean 6MWT greater than or equal 300 meters and less than or equal to 450 meters
• Stable pulmonary function: forced vital capacity (FVC) equal to or greater than 50%
predicted
Exclusion Criteria:
• Treatment with gene therapy at any time
• Previous treatment with SMT C1100, PRO045 (BMN 045), PRO053 (BMN 053) or PRO051 (BMN
051) within 24 weeks prior to Week 1
• Current or previous treatment with any other experimental treatment (other than
deflazacort) within 12 weeks prior to Week 1
• Major surgery within 3 months prior to Week 1
• Presence of other clinically significant illness
Other inclusion/exclusion criteria may apply
1. Subjects with a diagnosis of iobenguane avid, relapsed, high-risk neuroblastoma based
on revised INRC criteria who have completed at least one cycle of induction and
consolidation therapy with an INRC criterion of partial response or better, and then
showed new progressive disease (revised INRC criteria progressive disease) as
described in Park, et al. (2017).This may include one or more of the following drugs:
cyclophosphamide or ifosfamide, cisplatin or carboplatin, vincristine, doxorubicin
(adriamycin), etoposide, topotecan, and/or busulfan and melphalan (sometimes used
during stem cell transplant) and/or immunotherapy. (If a subject is symptomatic and
for logistical reasons cannot be treated immediately with 131I-MIBG, 1 to 2 cycles of
"bridging chemotherapy" or immunotherapy will be permitted. If "bridging chemotherapy"
or immunotherapy is applied, approximately 4 weeks will be required for reassessment
of the baseline including tumor assessment.
2. Must be therapeutic 131I-MIBG naive.
3. All soft tissue lesions identified on CT/MRI scans must be iobenguane-avid lesions on
an iobenguane (123I) scan or any non iobenguane avid lesions biopsy proven to be
non-tumor lesions.
4. Adequate cryopreserved autologous peripheral blood stem cells or bone marrow (at least
2 aliquots of 2.0 × 10exp6 CD34/kg at the time of study enrollment).
5. If a man, must agree to use an adequate contraception method as deemed appropriate by
the Investigator (e.g., vasectomy, condoms) or partner using effective contraception
and to not donate sperm during the study and for 90 days after receiving the last dose
of study drug.
6. If a woman of childbearing potential, have a negative serum pregnancy test result
prior to each dosing and, if sexually active, be practicing an effective method of
birth control [e.g., intrauterine device, double-barrier method (i.e., diaphragm, or a
cervical cap) with intravaginal spermicidal foam, cream or gel], or male partner
sterilization throughout the study.
7. Age at study entry ≥1 year.
8. Previous platelet transfusions are permitted, as long as the subject has a platelet
count ≥50,000/μL without transfusion support for at least 1 week.
9. Subjects must have a minimum pulse oximetry measurement of at least 94% at baseline.
10. An absolute neutrophil count ≥750/μL without growth factor for 5 days.
11. Liver function parameter results: total bilirubin ≤1.5 × upper limit of normal for
age, and serum glutamic-pyruvic transaminase (alanine aminotransferase) [SGPT (ALT)]
and serum glutamic-oxaloacetic transaminase (aspartate aminotransferase) [SGOT (AST)]
<3 × upper limit of normal (note that for ALT, the upper limit of normal for all sites
is defined as 45 U/L).
12. Normal thyroid function as measured by T4 and TSH or have abnormal results that are
not considered clinically important by the Investigator or may be receiving
levothyroxine.
13. Cardiac Function: Ejection fraction (≥55%) documented by echocardiogram within 1 month
prior to Visit 1 (baseline).
14. Karnofsky Performance Status (for subjects >16 years of age) or the Lansky Performance
Status Performance Status (for subjects 1 to 16 years of age) ≥50%.
15. Full recovery from the toxic effects of any prior therapy.
Exclusion Criteria:
1. Evidence of non-avid iobenguane lesions on iobenguane (123I) scan including soft
tissue disease on CT/MRI that is not iobenguane-avid.
2. Subjects with primary refractory disease.
3. Subjects within 5 half-lives after any antibody-based immunotherapy, or have not
recovered from effects of any biologic therapy.
4. Subjects that are refractory to the prior treatment regimen.
5. Subjects <12 weeks after myeloablative therapy with autologous stem cell transplant.
6. Subjects who have had an allogeneic stem cell treatment less than 4 months from Visit
1 are excluded. Those who have received allogeneic stem cell treatment more than 4
months from Visit 1 must have recovered and have no active graft versus host disease
(GVHD) to be eligible.
7. History of local radiation therapy within the last 3 months.
8. History of total body irradiation.
9. Subjects do not have adequate renal function defined as adjusted serum creatinine ≥1.5
× upper limit of normal for sex and age.
10. Subjects who are on hemodialysis.
11. Pregnancy or breastfeeding.
12. Significant active infections including active hepatitis B, or hepatitis C infection,
or known infection with human immunodeficiency virus (HIV) (testing for HIV is not
required prior to study entry).
13. Clinically important cardiac, pulmonary, and hepatic impairment.
Drug: 131I-MIBG
Neuroblastoma, Neoplasms, Neuroectodermal Tumors, Brain and Nervous System
Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy (STRONG)
The purpose of this trial is to evaluate the safety and tolerability of intrathecal
administration of onasemnogene abeparvovec-xioi in infants and children with Spinal Muscular
Atrophy with 3 copies of SMN2 and deletion of SMN1.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Susan Iannaccone
13463
All
6 Months to 60 Months old
Phase 1
This study is NOT accepting healthy volunteers
NCT03381729
STU 062016-082
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Key Inclusion Criteria
• Patients ≥6 months and up to 60 months (1800 days) of age at time of dosing following
diagnostic confirmation during screening period by genotype who demonstrate the
ability to sit unassisted for 10 or more seconds but cannot stand or walk
• Diagnostic confirmation by genotype includes lab documentation of homozygous absence
of SMN1 exon 7; with exactly three copies of SMN2
• Negative gene testing for SMN2 gene modifier mutation (c.859G>C)
• Onset of clinical signs and symptoms consistent with spinal muscular atrophy (SMA) at
< 12 months of age
• Able to sit independently and not standing or walking independently. Definition of
sitting independently is defined by the World Health Organization Multicentre Growth
Reference Study (WHO-MGRS) criteria of being able to sit up unsupported with head
erect for at least 10 seconds. Child should not use arms or hands to balance body or
support position (Wijnhoven 2004)
• Be up-to-date on childhood vaccines that include palivizumab prophylaxis (also known
as Synagis) to prevent respiratory syncytial virus (RSV) infections are also
recommended in accordance with American Academy of Pediatrics (AAP 2009)
Key Exclusion Criteria
• Current or historical ability to stand or walk independently
• Contraindications for spinal tap procedure or administration of intrathecal therapy or
presence of an implanted shunt for the drainage of CSF or an implanted central venous
(CNS) catheter
• Severe contractures as determined by designated Physical Therapist(s) at screening
that interfere with either the ability to attain/demonstrate functional measures or
interferes with ability to receive intrathecal (IT) dosing
• Severe scoliosis (defined as ≥ 50° curvature of spine) evident on X-ray examination
• Previous, planned or expected scoliosis repair surgery/procedure within 1 year of dose
administration
• Use of invasive ventilatory support (tracheotomy with positive pressure) or pulse
oximetry < 95% saturation at screening while the patient is awake, or for high
altitudes > 1000 m, oxygen saturation < 92% while the patient is awake
• Pulse oximetry saturation must not decrease ≥ four (4) percentage points between
screening and highest value on day of dosing
• Use or requirement of non-invasive ventilatory support for 12 or more hours daily over
the two (2) weeks prior to dosing
• Medical necessity for a gastric feeding tube, where the majority of feedings are given
by non-oral methods (i.e., nasogastric tube or nasojejunal tube) or patients whose
weight-for-age falls below the 3rd percentile based on WHO Child Growth Standards
(Onis 2006). Placement of a permanent gastrostomy prior to screening is not an
exclusion
• Use or requirement of non-invasive ventilatory support for 12 or more hours daily over
the two (2) weeks prior to dosing
• Medical necessity for a gastric feeding tube, where the majority of feedings are given
by non-oral methods or patients whose weight-for-age falls below the 3rd percentile
based on WHO Child Growth Standards (Onis 2006). Placement of a permanent gastrostomy
prior to screening is not an exclusion
• Active viral infection (includes human immunodeficiency virus (HIV) or serology
positive for hepatitis B or C, or Zika virus)
• Serious non-respiratory tract illness requiring systemic treatment and/or
hospitalization within two (2) weeks prior to study entry
• Respiratory infection requiring medical attention, medical intervention or increase in
supportive care of any manner within four (4) weeks prior to study entry
• Severe non-pulmonary/respiratory tract infection within four (4) weeks before study
dosing or concomitant illness that in the opinion of the Principal Investigator (PI)
creates unnecessary risks for gene transfer such as:
• Major renal or hepatic impairment
• Known seizure disorder
• Diabetes mellitus
• Idiopathic hypocalciuria
• Symptomatic cardiomyopathy
• History of bacterial meningitis or brain or spinal cord disease, including tumors, or
abnormalities by magnetic resonance imaging (MRI) or computerized tomography (CT) that
would interfere with the lumbar puncture (LP) procedures or CSF circulation
• Known allergy or hypersensitivity to prednisolone or other glucocorticosteroids or
their excipients
• Known allergy or hypersensitivity to iodine or iodine-containing products
• Concomitant use of any of the following: drugs for treatment of myopathy or
neuropathy, agents used to treat diabetes mellitus, or ongoing immunosuppressive
therapy, plasmapheresis, immunomodulators such as adalimumab, or immunosuppressive
therapy within 3 months of study dosing
• Inability to withhold use of laxatives or diuretics in the 24 hours prior to dose
administration
• Anti-AAV9 antibody titers >1:50 as determined by Enzyme-linked Immunosorbent Assay
(ELISA) binding immunoassay
• Should a potential patient demonstrate anti AAV9 antibody titer > 1:50, he or she
may receive retesting within 30 days of the screening period and will be eligible
to participate if the anti AAV9 antibody titer upon retesting is ≤ 1:50
• Clinically significant abnormal laboratory values (GGT, ALT, and AST, or total
bilirubin > 2 × ULN, creatinine ≥ 1.0 mg/dL, hemoglobin [Hgb] < 8 or > 18 g/dL; white
blood cell [WBC] > 20,000 per cmm) prior to gene replacement therapy. Patients with an
elevated bilirubin level that is unequivocally the result of neonatal jaundice shall
not be excluded
• Participation in recent SMA treatment clinical trial or receipt of an investigational
or approved compound product or therapy received with the intent to treat SMA at any
time prior to screening for this study
• Oral beta agonists must be discontinued 30 days prior to dosing.
• Inhaled albuterol specifically prescribed for the purposes of respiratory
(bronchodilator) management is acceptable and not a contraindication at any time
prior to screening for this study
• Expectation of major surgical procedures during the 1-year study assessment period
A Study of Galcanezumab (LY2951742) in Participants 6 to 17 Years of Age With Episodic Migraine (REBUILD-1)
The main purpose of this study is to evaulate the efficacy and safety of galcanezumab in
participants 6 to 17 years of age for the preventive treatment of episodic migraine. The
primary objective is to demonstrate the superiority of galcanezumab versus placebo in the
reduction of monthly migraine headache days across the 3-month double-blind treatment period.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Eric Remster
150068
All
6 Years to 17 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03432286
STU 072018-085
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Inclusion Criteria:
• Have a diagnosis of migraine with or without aura as defined by the IHS ICHD-3
guidelines (1.1 or 1.2 according to ICHD-3 [2018]), with a history of migraine
headaches of at least 6 months prior to screening.
Exclusion Criteria:
• Participants who are taking, or are expected to take, therapeutic antibodies during
the course of the study (adalimumab, infliximab, trastuzumab, bevacizumab, etc.).
Prior use of therapeutic antibodies, other than antibodies to calcitonin gene-related
peptide (CGRP) or its receptor, is allowed if that use was more than 12 months prior
to baseline.
• Known hypersensitivity to multiple drugs, monoclonal antibodies or other therapeutic
proteins, or to galcanezumab or its excipients.
• Current use or prior exposure to galcanezumab, another CGRP antibody, or CGRP receptor
antibody, including those who have previously completed or withdrawn from this study
or any other study investigating a CGRP antibody.
• History of persistent daily headache, cluster headache or migraine subtypes including
hemiplegic (sporadic or familial) migraine and migraine with brainstem aura
(previously basilar-type migraine) as defined by IHS ICHD-3.
• History of significant head or neck injury within 6 months prior to screening; or
traumatic head injury at any time that is associated with significant change in the
quality or frequency of their headaches, including new onset of migraine following
traumatic head injury.
• Participants with a known history of intracranial tumors or developmental
malformations including Chiari malformations.
A Study of RVT-1401 in Myasthenia Gravis (MG) Patients
The purpose of the current study is to assess safety/tolerability and key pharmacodynamic
(PD) effects that are considered to be associated with clinical benefit (reduction of total
IgG and anti-AChR-IgG) in Myasthenia Gravis patients following treatment with RVT-1401 (also
known as IMVT-1401) compared to placebo.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Shaida Khan
137363
All
18 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT03863080
STU-2019-0635
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Inclusion Criteria:
1. Male or female ≥ 18 years of age.
2. Myasthenia Gravis Foundation of America (MGFA) Class II-IVa and likely not in need of
a respirator for the duration of the study as judged by the Investigator.
3. QMG score ≥12 at Screening and Baseline.
Other, more specific inclusion criteria are defined in the protocol.
Exclusion Criteria:
1. Use of rituximab, belimumab, eculizumab or any monoclonal antibody for
immunomodulation within 6 months prior to first dosing.
2. Immunoglobulins given by SC, IV (IVIG), or intramuscular route, or
plasmapheresis/plasma exchange (PE) within 4 weeks before Screening.
3. Thymectomy performed < 12 months prior to screening.
4. Total IgG level <6 g/L (at screening).
5. Absolute neutrophil count <1500 cells/mm3(at screening).
Other, more specific exclusion criteria are defined in the protocol
Neuromuscular Blockade in Patients With Severe Renal Impairment
This study is intended to be a single-site, prospective, randomized, double-blinded study
that intends to enroll a total of 60 patients with severe renal impairment undergoing surgery
with general endotracheal anesthesia at Parkland Hospital. Patients will be randomized to
receive either neostigmine (for reversal of cisatracurium) or sugammadex (for reversal of
rocuronium). A standardized anesthetic protocol that is usual and customary for the type of
operation the patient is having will be provided to the anesthesia teams of enrolled
subjects. The remainder of the anesthetic care of the subject will not deviate from the
standard of care. All patients will be monitored with continuous pulse oximetry
postoperatively for 24 hours.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Tiffany Moon
66760
All
18 Years to 80 Years old
Phase 2
This study is also accepting healthy volunteers
NCT03904550
STU-2018-0411
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Inclusion Criteria:
• 18-80 years old
• Severe renal impairment (CrCl < 30 mL/min)
• Undergoing non-emergent surgery that requires neuromuscular blockade
• Planned extubation in the operating room immediately after surgery
• American Society of Anesthesiologists (ASA) physical status classification 3 to 4
• Willing and able to consent in English or Spanish
• No personal history of neuromuscular disease
Exclusion Criteria:
• Age less than 18 or older than 80
• Patient does not speak English or Spanish
• Planned postoperative intubation/ventilation
• Allergy to sugammadex, neostigmine, glycopyrrolate, cisatracurium, or rocuronium
• Family or personal history of malignant hyperthermia
• Patient refusal
• Pregnant or nursing women
• "Stat" (emergent) cases
• Pre-existing muscle weakness of any etiology
• Patients on toremifene (a selective estrogen receptor modulator)
• Women on oral contraceptives who do not wish to use a non-hormonal method of
contraception for 7 days following surgery
A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients
MPS IIIA, also known as Sanfilippo A, is an inherited lysosomal storage disease (LSD). MPS
IIIA is caused by a deficiency in sulfamidase, one of the enzymes involved in the lysosomal
degradation of the glycosaminoglycan (GAG) heparan sulfate (HS). The natural course of MPS
IIIA is characterized by devastating neurodegeneration with initially mild somatic
involvement. The aim of the present study is to assess the safety, tolerability and efficacy
of long-term SOBI003 treatment. SOBI003 is a chemically modified recombinant human (rh)
Sulfamidase developed as an enzyme replacement therapy (ERT).
Call 214-648-5005 studyfinder@utsouthwestern.edu
Luis Umana Franco
150802
All
18 Months to 78 Months old
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT03811028
STU-2019-0796
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Inclusion Criteria:
• Completion of study SOBI003-001
• Informed consent obtained from the patient´s legally authorized representative
Exclusion Criteria:
• If, in the opinion of the investigator, there are patient specific safety concerns
that contraindicates further treatment with SOBI003
Dabrafenib Combined With Trametinib After Radiation Therapy in Treating Patients With Newly-Diagnosed High-Grade Glioma
This phase II trial studies how well the combination of dabrafenib and trametinib works after
radiation therapy in children and young adults with high grade glioma who have a genetic
change called BRAF V600 mutation. Radiation therapy uses high energy rays to kill tumor cells
and reduce the size of tumors. Dabrafenib and trametinib may stop the growth of tumor cells
by blocking BRAF and MEK, respectively, which are enzymes that tumor cells need for their
growth. Giving dabrafenib with trametinib after radiation therapy may work better than
treatments used in the past in patients with newly-diagnosed BRAF V600-mutant high-grade
glioma.
• PRE-ENROLLMENT ELIGIBILITY SCREENING (STEP 0): Patient is suspected of having
localized newly-diagnosed HGG, excluding metastatic disease
• PRE-ENROLLMENT ELIGIBILITY SCREENING (STEP 0): Patient and/or their parents or legal
guardians have signed informed consent for eligibility screening on APEC14B1 Part A.
• PRE-ENROLLMENT ELIGIBILITY SCREENING (STEP 0): Specimens obtained at the time of
diagnostic biopsy must be submitted through APEC14B1 as soon as possible (ASAP),
preferably within 13 calendar days of the procedure.
• Patients must be >= 3 years and =< 21 years of age at the time of enrollment
• Patients must have eligibility confirmed by Rapid Central Pathology and Molecular
Screening Reviews performed on APEC14B1
• Newly diagnosed high-grade glioma with BRAFV600-mutation
• Positive or negative results for H3 K27M by immunohistochemistry (IHC)
• Histologically confirmed high-grade glioma (World Health Organization [WHO] grade
III or IV) including but not limited to: anaplastic astrocytoma (AA), anaplastic
pleomorphic xanthoastrocytoma (aPXA), anaplastic gangliogliomas (aGG),
glioblastoma (GB), and high-grade astrocytoma, not otherwise specified (NOS)
• Patients must have had histologic verification of a high-grade glioma diagnosis.
Cerebrospinal fluid (CSF) cytology by lumbar puncture must be done if clinically
indicated and determined to be safe prior to study enrollment. If cytology proves
positive, the patient would be considered to have metastatic disease and would,
therefore, be ineligible.
• A pre- and post-operative brain magnetic resonance imaging (MRI) with and without
contrast and a baseline spine MRI with contrast must be obtained prior to enrollment.
The requirement for a post-operative MRI is waived for patients who undergo biopsy
only. If the spine MRI is positive, the patient would be considered to have metastatic
disease and would be ineligible.
• 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.
• Peripheral absolute neutrophil count (ANC) >= 1000/uL (within 7 days prior to
enrollment).
• Platelet count >= 100,000/uL (transfusion independent) (within 7 days prior to
enrollment).
• Hemoglobin >= 8.0 g/dL (may receive red blood cell [RBC] transfusions) (within 7 days
prior to enrollment).
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70
mL/min/1.73 m^2 (within 7 days prior to enrollment) or
• A serum creatinine based on age/gender as follows (within 7 days prior to enrollment):
• Age 3 to < 6 years (Male 0.8 mg/dL, Female 0.8 mg/dL)
• Age 6 to < 10 years (Male 1 mg/dL, Female 1 mg/dL)
• Age 10 to < 13 years (Male 1.2 mg/dL, Female 1.2 mg/dL)
• Age 13 to 16 < years (Male 1.5 mg/dL, Female 1.4 mg/dL)
• Age >= 16 years (Male 1.7 mg/dL, Female 1.4 mg/dL)
• Total bilirubin =< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to
enrollment), and
• Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135
U/L (within 7 days prior to enrollment). For the purpose of this study, the ULN for
SGPT is 45 U/L.
• Patients with a seizure disorder may be enrolled if their seizures are well controlled
while on non-enzyme inducing anticonvulsants permitted on this study.
• Patients must be enrolled and protocol therapy must be projected to begin no later
than 31 days after definitive surgery (day 0). If a biopsy only was performed, the
biopsy date will be considered the date of definitive surgery. For patients who have a
biopsy or incomplete resection at diagnosis followed by additional surgery, the date
of the last resection will be considered the date of definitive surgery.
• All patients and/or their parents or legal guardians must sign a written informed
consent.
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute
(NCI) requirements for human studies must be met.
Exclusion Criteria:
• Patients with intrinsic brainstem or primary spinal cord tumors will be excluded.
• Patients with metastatic disease (defined as neuraxis dissemination either by imaging
or by cytology) will be excluded.
• Patients must not have received any prior tumor-directed therapy including
chemotherapy, radiation therapy, immunotherapy, or bone marrow transplant for the
treatment of HGG other than surgical intervention and/or corticosteroids.
• Previous treatment with dabrafenib or another RAF inhibitor, trametinib or another MEK
inhibitor, or an ERK inhibitor.
• Patients with a history of a malignancy with confirmed activating RAS mutation.
• History of allergic reactions attributed to compounds of similar chemical or biologic
composition to dabrafenib, trametinib, and their excipients.
• Uncontrolled medical conditions (e.g., diabetes mellitus, hypertension, liver disease,
or uncontrolled infection), psychological, familial, sociological, or geographical
conditions that do not permit compliance with the protocol; or unwillingness or
inability to follow the procedures required in the protocol.
• Presence of active gastrointestinal (GI) disease or other condition (e.g., small bowel
or large bowel resection) that will interfere significantly with the absorption of
drugs.
• History of hepatitis B virus, or hepatitis C virus infection (patients with laboratory
evidence of cleared hepatitis B virus and/or hepatitis C virus may be enrolled).
• History or current diagnosis of cardiac disease indicating significant risk of safety
for patients participating in the study such as uncontrolled or significant cardiac
disease, including any of the following:
• Recent myocardial infarction (within the last 6 months);
• Uncontrolled congestive heart failure;
• Unstable angina (within last 6 months);
• Clinically significant (symptomatic) or known, uncontrolled cardiac arrhythmias
(e.g., sustained ventricular tachycardia, and clinically significant second or
third degree atrioventricular [AV] block without a pacemaker) except sinus
arrhythmia within the past 24 weeks prior to the first dose of study treatment;
• Coronary angioplasty or stenting (within last 6 months);
• Intra-cardiac defibrillators;
• Abnormal cardiac valve morphology (>= grade 2) documented by echocardiogram.
• Patients with a history or current evidence of retinal vein occlusion (RVO) or central
serous retinopathy (CSR), or predisposing factors to RVO or CSR (e.g., uncontrolled
glaucoma or ocular hypertension).
• Patients with presence of interstitial lung disease or pneumonitis.
• Female patients who are pregnant are ineligible since there is yet no available
information regarding human fetal or teratogenic toxicities.
• Lactating females are not eligible unless they have agreed not to breastfeed their
infants for the duration of the study and for 4 months following discontinuation of
study therapy.
• Female patients of childbearing potential are not eligible unless a negative pregnancy
test result has been obtained.
• Sexually active patients of reproductive potential (male or female) are not eligible
unless they have agreed to use an effective contraceptive method for the duration of
their study participation and for 4 months following discontinuation of study therapy.
Male patients (including those who have had a vasectomy) taking dabrafenib and
trametinib combination therapy must use a condom during intercourse while on study and
for 16 weeks after stopping treatment, and should not father a child during these
periods. Women of childbearing potential should use effective non-hormonal
contraception during therapy and for 4 weeks following discontinuation of dabrafenib
and at least 4 months following the last dose of trametinib in patients taking
combination therapy. Women should be advised that dabrafenib may decrease the efficacy
of hormonal contraceptives and an alternate method of contraception, such as barrier
methods, should be used.
Cladribine Tablets After Treatment With Natalizumab (CLADRINA)
The purpose of this study is to generate hypotheses regarding the safety, efficacy, and
immunological impact of cladribine tablets after treatment with natalizumab in patients with
relapsing-remitting multiple sclerosis (RRMS) and active secondary progressive multiple
sclerosis (active SPMS).
Call 214-648-5005 studyfinder@utsouthwestern.edu
Olaf Stuve
58631
All
18 Years to 60 Years old
Phase 4
This study is NOT accepting healthy volunteers
NCT04178005
STU-2019-1618
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Inclusion Criteria:
Patients who meet the following inclusion criteria will be eligible for enrollment in the
study:
1. Age between 18 and 60 years, inclusive.
2. Diagnosis of relapsing forms of MS, to include RRMS and active SPMS, diagnosed with
McDonald Criteria 2005, 2010, and/or 2017 (1-3)
3. EDSS 0 •5.5 (Functional system changes in cerebral (or mental) functions and in bowel
and bladder functions not used in determining EDSS for protocol eligibility).
4. Has had a minimum of 12 months of continuous natalizumab therapy (300 mg/d), including
patients receiving extended interval dosing of natalizumab (e.g., less frequently than
every-4-week infusion).
5. Negative history for any relapses at least 28 days prior to enrollment.
6. Weighing between 40 kilograms or more.
7. Female subjects of childbearing potential must use effective methods of contraception
to prevent pregnancy for 4 weeks before initiation of cladribine tablets and must
agree to continue to practice adequate contraception for at least 6 months after the
last dose. Women using systemically acting hormonal contraceptives should add a
barrier method during cladribine treatment and for at least 4 weeks after the last
dose in each treatment year.
8. Female subjects must not be pregnant; female subjects must not be lactating or
breast-feeding at least 10 days after the last dose.
9. Male subjects must be willing to use a condom during dosing and for six months after
the last dose. Alternatively, their female partner must use another form of
contraception (such as an intra-uterine device [IUD], barrier method with spermicide,
or hormonal contraceptive [e.g., implant, injectable, patch or oral]) during dosing
and for six months after last dose.
10. Understands and is capable of following through with study protocol requirements and
assessments.
11. Willing to provide voluntary and informed consent based on the Health Insurance
Portability and Accountability Act (HIPPA).
Exclusion Criteria:
Patients who meet any of the following exclusion criteria will not be eligible for
enrollment in the study:
1. Natalizumab failure based on clinician's discretion.
2. Not active progressive MS (4).
3. A diagnosis of PML or any suspicion of PML.
4. A diagnosis of Clinically Isolated Syndrome
5. Known hypersensitivity to cladribine.
6. Any prior exposure to cladribine.
7. Lymphocyte count not within normal limits of the local, hospital laboratory.
8. Previous or current exposure to mitoxantrone, azathioprine, methotrexate,
cyclophosphamide, myelosuppressive treatments, total lymphoid irradiation.
9. Receiving oral or systemic corticosteroid treatments within the 28 days prior to
enrollment.
10. Receiving cytokine base treatment, Intra Venous Immuno Globulin (IVIG) or Plasma
pheresis, 3 months prior to enrollment in the study.
11. Having platelet count or neutrophil count below the lower limit of the normal range
within the 28 days prior to enrollment in the study.
12. Positive for HIV, or positive hepatitis C antibody test or hepatitis B surface antigen
test and/or core antibody test for IgG and/or IgM.
13. History of tuberculosis (TB), presence of active tuberculosis, or latent tuberculosis
as detected by local standard of practice like imaging (e.g., chest X-ray, chest CT
scan, MRI) and/or positive QuantiFERON-TB Gold test and/or skin test and/or clinical
examination or has had latent TB disease at any time in the past.
14. Immunocompromised subjects, including subjects currently receiving immunosuppressive
or myelosuppressive therapy with, e.g., monoclonal antibodies, methotrexate,
cyclophosphamide, cyclosporine or azathioprine, or chronic use of corticosteroids.
15. Active malignancy or history of malignancy.
16. Received a live vaccine within 6 weeks prior to cladribine tablet administration or
intends to receive a live vaccination during the trial. After the last dose of
cladribine tablets, the subject should avoid live vaccine as long as the subject's
white blood cell counts are not within normal limits.
17. Allergy or hypersensitivity to gadolinium and/or any other contraindication to perform
an MRI.
18. Has any renal condition that would preclude the administration of gadolinium (e.g.
acute or chronic severe renal insufficiency (GFR < 30 mL/min/1.73m2)
-
31P-MRS Imaging to Assess the Effects of CNM-Au8 on Impaired Neuronal Redox State in Multiple Sclerosis (REPAIR-MS)
REPAIR-MS is a single-center open label, sequential group, investigator and patient blinded
study to assess the CNS metabolic effects, safety, pharmacokinetics, and pharmacodynamics of
CNM-Au8 in patients who have been diagnosed with Multiple Sclerosis (MS) within fifteen (15)
years of Screening. The primary endpoint is the ratio of the oxidized to reduced form of
nicotinamide adenine dinucleotide (NAD+:NADH) measured non-invasively by 31phosphorous
magnetic resonance spectroscopy (31P-MRS).
Call 214-648-5005 studyfinder@utsouthwestern.edu
Benjamin Greenberg
105091
All
18 Years to 55 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03993171
STU-2019-0992
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Inclusion Criteria:
1. At least 18 years of age and up to 55 years (inclusive) of age at Baseline.
2. Clinical diagnosis of Multiple Sclerosis (MS) who have had Relapsing MS (RMS) no
longer than 15 years from diagnosis.
3. Stable treatment with natalizumab for at least the prior six (6) months.
4. Stable disease activity over the prior six (6) months.
5. All hematological parameters and biochemical parameters deemed stable or transient in
nature.
6. Able to understand and give written informed consent.
Exclusion Criteria:
1. Patients with a clinical relapse requiring systemic steroid treatment within the prior
six (6) months.
2. Patients treated with any other MS therapy other than natalizumab; or treated with
clemastine fumarate.
3. Patients with a history of significant other major medical condition that may
interfere with the conduct of the study or interpretation of the study results.
4. Patients who may have difficulty complying with the protocol and/or study procedures.
5. Patient with clinically significant abnormalities in hematology, blood chemistry, ECG,
or physical examination not resolved by the Baseline visit which according to
Investigator can interfere with study participation.
6. Patients with clinically significant hepatic or renal dysfunction or clinical
laboratory findings that would limit the interpretability of change in liver or kidney
function, or those with low platelet counts (< 150 x 109 per liter) or eosinophilia
(absolute eosinophil count of ≥500 eosinophils per microliter).
7. Patients with a prior history of, or positive serological assay for the presence of
HIV infection, or laboratory evidence of active or chronic infection with hepatitis C
(HCV) or hepatitis B (HBV).
8. Patients participating in any other investigational drug trial or using an
investigational drug (within 12 weeks prior to screening and thereafter)
9. Positive screen for drugs of abuse or known alcohol abuse.
10. Females who are pregnant, have a positive pregnancy test, are nursing, or who plan to
get pregnant during the course of this clinical trial or within 6 months of the end of
this trial.
11. Women of child-bearing potential, or men, who are unwilling or unable to use accepted
methods of birth control during the study and for 6 months following completion of
study participation.
12. Patients with implanted metal objects in their body that may be affected by an MRI
procedure.
13. Patients who are claustrophobic or otherwise unlikely to be able to complete the MRI
scanning procedures.
14. Patients with a history of gold allergy.
15. Patient is considered a suicide risk in the opinion of the Investigator, has
previously made a suicide attempt, or is currently demonstrating active suicidal
ideation.
16. Any active ophthalmological cause for retinal damage other than MS or based on the
Investigator's judgment any other ophthalmic diseases that would confound the study
results or optical coherence tomography assessment.
17. PRN use of stimulant medications including: amphetamine, dextroamphetamine,
lisdexamfetamine, methylphenidate, or modafinil.
Drug: gold nanocrystals
Relapsing Remitting Multiple Sclerosis, Brain and Nervous System
Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D
To explore triheptanoin (C7 oil) compatibility with the ketogenic diet by evaluating EEG, and
seizure rate, glycemia and ketosis in proven G1D patients receiving a ketogenic diet.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Juan Pascual
85158
All
30 Months to 35 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03301532
STU 102015-091
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Inclusion Criteria:
• Diagnosis of glucose transporter type 1 deficiency (G1D), confirmed by clinical
genotyping at a CLIA-certified laboratory.
• Stable on ketogenic diet at 2.5:1 to 4:1 ratio (i.e., no changes in ratio will have
taken place for 2 months). The initiation of a ketogenic diet is previous to •and
thus is not part of this study.
• Males and females 30 months to 35 years and 11 months old inclusive.
Exclusion Criteria:
• Subjects with evidence of independent, unrelated metabolic and/or genetic disease.
• Subjects with a chronic gastrointestinal disorder, such as irritable bowel syndrome,
crohn's disease, or colitis that could increase the subject's risk of developing
diarrhea or stomach pain.
• Subjects with a BMI (body mass index) greater than or equal to 30.
• Subjects currently not on ketogenic diet.
• Women who are pregnant or breast feeding may not participate. Women who plan to become
pregnant during the course of the study, or who are unwilling to use birth control to
prevent pregnancy (including abstinence) may not participate. Females age 10 and over
will be asked to provide a urine sample for a pregnancy test via dipstick. Subjects
will be asked to agree to abstinence or another form of birth control for the duration
of the study.
• Allergy/sensitivity to C7
• Previous use of triheptanoin less than 1 month prior to study initiation.
• Treatment with medium chain triglycerides in the last 24 hours.
• Subjects exhibiting signs of dementia, or diagnosed with any degenerative brain
disorder (such as Alzheimer's disease) that would confound assessment of cognitive
changes, in the opinion of the investigator.
• Active drug or alcohol use or dependence that, in the opinion of the investigator,
would interfere with adherence to study requirements.
• Inability or unwillingness of subject or legal guardian/representative to give written
informed consent, or assent for children age 10-17,
• Addition of a new antiseizure drug in the previous 3 months.
Response and Biology-Based Risk Factor-Guided Therapy in Treating Younger Patients With Non-high Risk Neuroblastoma
This phase III trial studies how well response and biology-based risk factor-guided therapy
works in treating younger patients with non-high risk neuroblastoma. Sometimes a tumor may
not need treatment until it progresses. In this case, observation may be sufficient.
Measuring biomarkers in tumor cells may help plan when effective treatment is necessary and
what the best treatment is. Response and biology-based risk factor-guided therapy may be
effective in treating patients with non-high risk neuroblastoma and may help to avoid some of
the risks and side effects related to standard treatment.
• Patients must be:
• < 12 months (< 365 days) of age at diagnosis with INRG stage L1; or
• < 18 months (< 547 days) of age at diagnosis with INRG stage L2 or stage Ms
neuroblastoma/ganglioneuroblastoma
• Enrollment on ANBL00B1 or APEC14B1 is required for all newly diagnosed patients
• Patients must have newly diagnosed v-myc avian myelocytomatosis viral oncogene
neuroblastoma derived homolog (MYCN) non-amplified neuroblastoma (International
Classification of Diseases for Oncology [ICD-O] morphology 9500/3) or MYCN
non-amplified ganglioneuroblastoma verified by histology
• Patients must meet the specified criteria for one of the treatment groups defined
below; genomic features include MYCN gene amplification, segmental chromosome
aberrations (somatic copy number loss at 1p, 3p, 4p, or 11q or somatic copy number
gain at 1q, 2p, or 17q) and deoxyribonucleic acid (DNA) index
• "Favorable" genomic features are defined by one or more whole-chromosome gains or
hyperdiploid tumor (DNA index > 1) in the absence of segmental chromosome
aberrations as defined above
• "Unfavorable" genomic features are defined by the presence of any segmental
chromosome aberration (somatic copy number loss at 1p, 3p, 4p, or 11q or somatic
copy number gain at 1q, 2p, or 17q) or diploid tumor (DNA index = 1); this
includes copy neutral loss of heterozygosity (LOH)
• Only patients with MYCN non-amplified tumors are eligible for this study
• Group A: patients < 12 months (< 365 days) of age with newly diagnosed INRG stage L1
neuroblastoma/ganglioneuroblastoma who meet the following criteria:
• Greatest tumor diameter < 5 cm of adrenal or non-adrenal origin
• Patients with non-adrenal primaries are eligible, but must have positive uptake
on metaiodobenzylguanidine (MIBG) scan or elevated catecholamine metabolites
(urine or serum) to support the diagnosis of neuroblastoma
• No prior tumor resection or biopsy
• Group A will be further split into two subsets, which are mutually exclusive, for
statistical purposes
• Group A1:
• > 6 months and < 12 months of age with an adrenal primary tumor < 5 cm in
greatest diameter OR
• Patients less than 6 months of age with an adrenal primary tumor > 3.1 and <
5 cm in greatest diameter OR
• < 12 months of age with a non-adrenal primary site < 5 cm in greatest
diameter
• Group A2: =< 6 months of age with an adrenal primary site and tumor =< 3.1 cm in
greatest diameter.
• Group B: patients < 18 months (< 547 days) of age with newly diagnosed INRG stage L2
neuroblastoma/ganglioneuroblastoma who meet the following criteria:
• No life threatening symptoms or no impending neurologic or other organ function
compromise (e.g. epidural or intraspinal tumors with existing or impending
neurologic impairment, periorbital or calvarial-based lesions with existing or
impending cranial nerve impairment, anatomic or mechanical compromise of critical
organ function by tumor [abdominal compartment syndrome, urinary obstruction,
etc.]); horner syndrome is not considered neurologic compromise
• No prior tumor resection, tumor biopsy ONLY
• Only patients with both favorable histology and favorable genomic features will
remain on study as part of Group B; the institution will be notified of
histologic and genomic results within 3 weeks of specimen submission on ANBL00B1
or APEC14B1
• Group C: patients < 18 months (< 547 days) of age with newly diagnosed INRG stage Ms
neuroblastoma/ganglioneuroblastoma
• No prior radiotherapy or chemotherapy, with the exception of dexamethasone, which is
allowed
• All patients and/or their parents or legal guardians must sign a written informed
consent
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute
(NCI) requirements for human studies must be met
Exclusion Criteria:
• Patients with MYCN amplified tumors are not eligible
• Group B and C patients who do not enroll on ANBL1232 within 4 weeks of definitive
diagnostic procedure
• Group A and C patients, not required to undergo tumor biopsy, who do not enroll on
ANBL1232 within 4 weeks of confirmatory imaging study
Computer Training Program for Younger Patients With a Brain Tumor Who Underwent Radiation Therapy
This randomized clinical trial studies how well an adaptive computerized cognitive training
program works compared to a non-adaptive computerized cognitive training program in treating
younger patients with brain tumor who underwent radiation therapy. Providing a computer
training program may improve the well-being and quality of life of patients with cognitive
(physical and mental) function difficulties caused by radiation therapy to the brain.
• Patient must be newly diagnosed or relapsed/progressed with a brain tumor that has not
previously been treated with CRT
• Note: COG therapeutic study participation is not required for ACCL10P1 enrollment
• Patient enrollment must occur within 4 calendar months following completion of CRT
• Reminder: after patient enrollment, baseline testing followed by randomization
must occur within 2-4 months after completion of CRT
• The patient must have an identified caregiver who is willing and able to oversee the
training practice during the intervention period (ie, for 5-9 weeks starting
approximately 3 months after completion of CRT)
• The patient must have access to a telephone and phone number where they can be reached
• The patient and caregiver must have reading, speaking and listening comprehension of
English
• All patients and/or their parents or legal guardians must sign a written informed
consent (patient assent is also recommended when applicable according to each
institution's policy)
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute
(NCI) requirements for human studies must be met
Exclusion Criteria:
• Patients with pontine glioma are not eligible
• Patients with an estimated survival of less than one year are not eligible
• Patients with a history of traumatic brain injury prior to tumor diagnosis are not
eligible
• Patients with a motor, visual, or auditory handicap that prevents computer use (e.g.,
unresolved posterior fossa syndrome) are not eligible to participate in this trial
• Patients with full-scale intelligence quotient (IQ) < 70 per previous testing OR
existing diagnosis of/educational classification as a student with an intellectual
disability are not eligible
Procedure: Cognitive Assessment, Other: Computer-Assisted Cognitive Training, Procedure: Psychosocial Assessment and Care, Other: Quality-of-Life Assessment, Other: Questionnaire Administration
Brain Neoplasm, Recurrent Brain Neoplasm, Brain and Nervous System
Primary Objective:
Long-term safety and pharmacokinetics (PK) of neoGAA
Secondary Objective:
Long-term effect of neo-GAA on pharmacodynamic and exploratory efficacy variables
Call 214-648-5005 studyfinder@utsouthwestern.edu
Jaya Trivedi
46764
All
Not specified
Phase 2/Phase 3
This study is NOT accepting healthy volunteers
NCT02032524
STU 012014-036
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Inclusion criteria:
Patients with Pompe disease who previously completed a neoGAA study. The patient and/or
their parent/legal guardian is willing and able to provide signed informed consent, and
the patient, if <18 years of age, is willing to provide assent if deemed able to do so.
The patient (and patient's legal guardian if patient is <18 years of age) must have the
ability to comply with the clinical protocol.
The patient, if female and of childbearing potential, must have a negative pregnancy test
[urine beta-human chorionic gonadotropin] at baseline.
Exclusion criteria:
The patient is concurrently participating in another clinical study using investigational
treatment.
The patient, in the opinion of the Investigator, is unable to adhere to the requirements
of the study.
The patient has clinically significant organic disease (with the exception of symptoms
relating to Pompe disease), including clinically significant cardiovascular, hepatic,
pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent
illness, or extenuating circumstance that, in the opinion of the Investigator, precludes
participation in the study or potentially decreases survival.
The above information is not intended to contain all considerations relevant to a
patient's potential participation in a clinical trial.
Difluoromethylornithine (DFMO) will be used in an open label, single agent, multicenter,
study for patients with neuroblastoma in remission. In this study subjects will receive 730
Days of oral difluoromethylornithine (DFMO) at a dose of 750 mg/m2 ± 250 mg/m2 BID (strata 1,
2, 3, and 4) OR 2500 mg/m2 BID (stratum 1B) on each day of study. This study will focus on
the use of DFMO in high risk neuroblastoma patients that are in remission as a strategy to
prevent recurrence.
• All patients must have a pathologically confirmed diagnosis of neuroblastoma, < 30.99
years of age and classified as high risk at the time of diagnosis. Exception: patients
who are initially diagnosed as non-high-risk neuroblastoma, but later converted
(and/or relapsed) to high risk neuroblastoma are also eligible.
• All patients must be in complete remission (CR):
1. No evidence of residual disease on scan
2. No evidence of disease metastatic to bone marrow.
• Specific Criteria by Stratum:
Stratum 1/1B: All patients must have completed standard upfront therapy that replicates
treatment which patients who were enrolled on ANBL0032 received, including:
intensive induction chemotherapy and (if feasible) resection of primary tumor, followed by:
consolidation with high-dose chemotherapy with stem cell transplant and radiotherapy,
followed by: immunotherapy with Ch14.18/IL-2/GM-CSF (dinutuximab) and retinoic acid;.
All subjects on Stratum 1/B must have also met the following criteria:
• A pre-transplant disease status evaluation that met International Neuroblastoma Response
Criteria (INRC) for CR (complete response), VGPR (very good partial response), or PR
(partial response) for primary site, soft tissue metastases and bone metastases. Patients
who meet those criteria must also meet the protocol-specified criteria for bone marrow
response prior to transplant as outlined below: No more than 10% tumor involvement (based
on total nucleated cellular content) seen on any specimen from a bilateral bone marrow
aspirate/biopsy.
Stratum 2: Neuroblastoma that is in first complete remission following standard upfront
therapy different from that described for Stratum 1.
Stratum 3: Neuroblastoma that failed to have a response of at least PR following induction
chemotherapy and surgical resection of the primary tumor, but that has achieved CR
following additional therapy.
Stratum 4: Patients who have achieved a second or subsequent CR following relapse(s).
• Pre-enrollment tumor survey: Prior to enrollment on this study, a determination of
mandatory disease staging must be performed:
• Tumor imaging studies including
• Bilateral bone marrow aspirates and biopsy
• This disease assessment is required for eligibility and preferably should be done
within 2 weeks prior to enrollment, but must be done within a maximum of 4 weeks
before enrollment.
• Timing from prior therapy:
Stratum 1/1B: Enrollment no later than 60 days after completion of upfront therapy, (last
dose of cis-retinoic acid) with a maximum of 6 cycles of cis-retinoic acid maintenance
therapy.
Stratum 2, 3 and 4: Enrollment no later than 60 days from last dose of the most recent
therapy.
• Patients must have a Lansky or Karnofsky Performance Scale score of > 50% and patients
must have a life expectancy of ≥ 2 months.
• All clinical and laboratory studies for organ functions to determine eligibility must
be performed within 7 days prior to enrollment unless otherwise indicated below.
• Patients must have adequate organ functions at the time of registration:
• Hematological: Total absolute phagocyte count ≥1000/μL
• Liver: Subjects must have adequate liver function
• Renal: Adequate renal function
• Females of childbearing potential must have a negative pregnancy test. Patients of
childbearing potential must agree to use an effective birth control method. Female
patients who are lactating must agree to stop breast-feeding.
• Written informed consent in accordance with institutional and FDA (food and drug
administration) guidelines must be obtained from all subjects (or patients' legal
representative).
Exclusion Criteria:
• BSA (Body Surface Area) of <0.25 m2.
• Investigational Drugs: Subjects who are currently receiving another investigational
drug are excluded from participation.
• Anti-cancer Agents: Subjects who are currently receiving other anticancer agents are
not eligible. Subjects must have fully recovered from hematological and bone marrow
suppression effects of prior chemotherapy.
• Infection: Subjects who have an uncontrolled infection are not eligible until the
infection is judged to be well controlled in the opinion of the investigator.
• Subjects who, in the opinion of the investigator, may not be able to comply with the
safety monitoring requirements of the study, or in whom compliance is likely to be
suboptimal, should be excluded.
A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
Brief Summary: This Phase IIb study is a randomized, double-blind, parallel group, placebo
and active-controlled study to evaluate the efficacy, safety, PD, and population PK of
vamorolone administered orally at daily doses of 2.0 mg/kg and 6.0 mg/kg versus prednisone
0.75 mg/kg/day and placebo over a Treatment Period of 24 weeks, and to evaluate persistence
of effect over a Treatment Period of 48 weeks in ambulant boys ages 4 to <7 years with DMD.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Diana Castro
102470
Male
4 Years to 7 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03439670
STU 032018-026
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Inclusion Criteria:
1. Subject's parent(s) or legal guardian(s) has (have) provided written informed consent
and Health Insurance Portability and Accountability Act (HIPAA) authorization, where
applicable, prior to any study-related procedures; participants will be asked to give
written or verbal assent according to local requirements
2. Subject has a centrally confirmed (by TRiNDS central genetic counselor[s]) diagnosis
of DMD as defined as:
• Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin
deficiency, and clinical picture consistent with typical DMD, OR
• Identifiable mutation within the DMD gene (deletion/duplication of one or more
exons), where reading frame can be predicted as 'out-of-frame,' and clinical
picture consistent with typical DMD, OR
• Complete dystrophin gene sequencing showing an alteration (point mutation,
duplication, other) that is expected to preclude production of the dystrophin
protein (i.e., nonsense mutation, deletion/duplication leading to a downstream
stop codon), with a clinical picture consistent with typical DMD;
3. Subject is ≥ 4 years and <7 years of age at time of enrollment in the study;
4. Subject weighs >13.0 kg and ≤ 39.9 kg at the Screening Visit;
5. Subject is able to walk independently without assistive devices;
6. Subject is able to complete the Time to Stand Test (TTSTAND) without assistance in <10
seconds, as assessed at the Screening Visit;
7. Clinical laboratory test results are within the normal range at the Screening Visit,
or if abnormal, are not clinically significant, in the opinion of the Investigator.
[Notes: Serum gamma glutamyl transferase (GGT), creatinine, and total bilirubin all
must be ≤ upper limit of the normal range at the Screening Visit. An abnormal vitamin
D level that is considered clinically significant will not exclude a subject from
randomization];
8. Subject has evidence of chicken pox immunity as determined by:
• Presence of IgG antibodies to varicella, as documented by a positive test result
from the local laboratory from blood collected during the Screening Period, OR
• Documentation, provided at the Screening Visit, that the subject has had 2 doses
of varicella vaccine, with or without serologic evidence of immunity; the second
of the 2 immunizations must have been given at least 14 days prior to
randomization.
9. Subject is able to swallow tablets, as confirmed by successful test swallowing of
placebo tablets during the Screening Period; and
10. Subject and parent(s)/guardian(s) are willing and able to comply with scheduled
visits, study drug administration plan, and study procedures.
Exclusion Criteria:
1. Subject has current or history of major renal or hepatic impairment, diabetes mellitus
or immunosuppression;
2. Subject has current or history of chronic systemic fungal or viral infections;
3. Subject has had an acute illness within 4 weeks prior to the first dose of study
medication;
4. Subject has used mineralocorticoid receptor agents, such as spironolactone,
eplerenone, canrenone (canrenoate potassium), prorenone (prorenoate potassium),
mexrenone (mexrenoate potassium) within 4 weeks prior to the first dose of study
medication;
5. Subject has a history of primary hyperaldosteronism;
6. Subject has evidence of symptomatic cardiomyopathy [Note: Asymptomatic cardiac
abnormality on investigation would not be exclusionary];
7. Subject is currently being treated or has received previous treatment with oral
glucocorticoids or other immunosuppressive agents [Notes: Past transient use of oral
glucocorticoids or other oral immunosuppressive agents for no longer than 1 month
cumulative, with last use at least 3 months prior to first dose of study medication,
will be considered for eligibility on a case-by-case basis, unless discontinued for
intolerance. Inhaled and/or topical glucocorticoids are permitted if last use is at
least 4 weeks prior to first dose of study medication or if administered at stable
dose beginning at least 4 weeks prior to first dose of study medication and
anticipated to be used at the stable dose regimen for the duration of the study];
8. Subject has an allergy or hypersensitivity to the study medication or to any of its
constituents;
9. Subject has used idebenone within 4 weeks prior to the first dose of study medication;
10. Subject has severe behavioral or cognitive problems that preclude participation in the
study, in the opinion of the Investigator;
11. Subject has previous or ongoing medical condition, medical history, physical findings
or laboratory abnormalities that could affect safety, make it unlikely that treatment
and follow-up will be correctly completed or impair the assessment of study results,
in the opinion of the Investigator;
12. Subject is taking (or has taken within 4 weeks prior to the first dose of study
medication) herbal remedies and supplements which can impact muscle strength and
function (e.g., Co-enzyme Q10, creatine, etc);
13. Subject is taking (or has taken within 3 months prior to the first dose of study
medication) any medication indicated for DMD, including Exondys51 and Translarna;
14. Subject has been administered a live attenuated vaccine within 14 days prior to the
first dose of study medication;
15. Subject is currently taking any other investigational drug or has taken any other
investigational drug within 3 months prior to the first dose of study medication;
16. Subject has a sibling who is currently enrolled in any vamorolone study or Expanded
Access Program, or who intends to enroll in any vamorolone study or Expanded Access
Program during the subject's participation in the VBP15-004 study; or
17. Subject has previously been enrolled in the study. Note: Any parameter/test may be
repeated at the Investigator's discretion during Screening to determine
reproducibility. In addition, subjects may be rescreened if ineligible due to a
transient condition which would prevent the subject from participating, such as an
upper respiratory tract infection or injury, or if ineligible due to negative
anti-varicella IgG antibody test result.
Efficacy of Ocrelizumab in Autoimmune Encephalitis
This pilot study is a randomized, double-blind, placebo controlled study of the efficacy of
ocrelizumab in autoimmune encephalitis. Subjects with new diagnosis of autoimmune
encephalitis will be invited to enroll in this study. Subjects will be randomized to receive
ocrelizumab (an anti-CD20 therapy) or matched placebo, and will undergo three infusions over
a six month period. Subjects will complete clinical visits over the study period, during
which safety monitoring and neuropsychological assessments will be performed to assess for
signs of clinical worsening from encephalitis. The primary outcome of this study is the
proportion of patients who fail to complete the twelve month period without clinical
worsening, as defined by the protocol. Subjects who experience early clinical worsening
during the study may be offered open-label treatment with ocrelizumab at the discretion of
the investigators.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Steven Vernino
67844
All
18 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT03835728
STU-2018-0185
Show full eligibility criteria
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Inclusion Criteria:
1. Age 18 or greater
2. Able to obtain informed consent from patient or appropriate designee
3. Possible autoimmune encephalitis as defined by Table 1:
1. Reasonable exclusion of alternative causes
2. Subacute onset (< 3 months) of memory deficits, altered consciousness, and/or
psychiatric symptoms
3. One or more of the following:
• CSF (cerebrospinal fluid) pleocytosis (>5 cells/µl corrected, if necessary,
for traumatic lumbar puncture)
• EEG (electroencephalogram) with epileptiform or focal slow wave
abnormalities involving temporal lobes
• Brain abnormalities on T2/FLAIR MRI restricted to the mesial temporal
(limbic) lobes
• Associated dyskinesias (faciobrachial dystonic movements or orofacial
dyskinesias)
4. Completed initial treatment with iv steroids (at least 3000mg solumedrol) and plasma
exchange (at least 3 exchanges) within the past 8 weeks
5. Presence of one (or more) of the following autoantibodies in serum or CSF
• NMDA receptor
• LGI1
• CASPR2
• DPPX
Exclusion Criteria:
1. Prior immunosuppression treatment in past year (other than steroids, intravenous
immunoglobulin and plasma exchange)
2. Active malignancy requiring chemotherapy
3. Pregnancy
4. Evidence of active hepatitis or tuberculosis infection
5. Medical condition that (in investigators opinion) precludes the use of ocrelizumab
Maximum Tolerated Dose, Safety, and Efficacy of Rhenium Nanoliposomes in Recurrent Glioma (ReSPECT)
This is a multi-center, sequential cohort, open-label, volume and dose escalation study of
the safety, tolerability, and distribution of 186RNL given by convection enhanced delivery to
patients with recurrent or progressive malignant glioma after standard surgical, radiation,
and/or chemotherapy treatment. The study uses a modified Fibonacci dose escalation, followed
by an expansion at the maximum tolerated dose (MTD) to determine efficacy. The starting
absorbed dose is 1mCi in a volume of 0.660mL.
1. At least 18 years of age
2. Ability to understand the purposes and risks of the study and has signed a written
informed consent form approved by the investigator's IRB/Ethics Committee
3. Histologically confirmed glioma
4. Progression by RANO criteria following standard treatment options with known survival
benefit (Temozolomide, Radiation, and Tumor Treating Fields [unless unwilling])
5. Patients who receive treatment with antiepileptic medications must have a two week
history of stable dose of antiepileptic without seizures prior to dosing
6. Patients with corticosteroid requirements to control cerebral edema must be maintained
at a stable or decreasing dose for a minimum of two weeks without progression of
clinical symptoms
7. A volume of enhancing tumor which falls within the treatment field volume being
evaluated in the respective cohort (see 4.1 Design)
8. ECOG performance status of 0 to 2
9. Life expectancy of at least 2 months
10. Acceptable liver function:
• Bilirubin ≤ 1.5 times upper limit of normal
• AST (SGOT) and ALT (SGPT) ≤ 3.0 times upper limit of normal (ULN);
11. Acceptable renal function:
• Serum creatinine ≤1.5xULN
12. Acceptable hematologic status (without hematologic support):
• ANC ≥1000 cells/uL
• Platelet count ≥100,000/uL
• Hemoglobin ≥9.0 g/dL
13. All women of childbearing potential must have a negative serum pregnancy test and male
and female subjects must agree to use effective means of contraception (surgical
sterilization or the use or barrier contraception with either a condom or diaphragm in
conjunction with spermicidal gel or an IUD) with their partner from entry into the
study through 6 months after the last dose
For part 2:
14. Bevacizumab naïve glioblastoma with no more than 1 recurrence
Exclusion Criteria:
1. The subject has evidence of acute intracranial or intratumoral hemorrhage either by
MRI or computerized tomography (CT) scan. Subjects with resolving hemorrhage changes,
punctate hemorrhage, or hemosiderin are eligible.
2. The subject is unable to undergo MRI scan (eg, has pacemaker).
3. The subject has not recovered to National Cancer Institute (NCI) Common Terminology
Criteria for Adverse Events (CTCAE) v4.0 Grade ≤ 1 from AEs (except alopecia, anemia
and lymphopenia) due to surgery, antineoplastic agents, investigational drugs, or
other medications that were administered prior to study.
4. The subject is pregnant or breast-feeding.
5. The subject has serious intercurrent illness, as determined by the treating physician,
that would compromise either patient safety or study outcomes such as:
• hypertension (two or more blood pressure readings performed at screening of > 150
mmHg systolic or > 100 mmHg diastolic) despite optimal treatment
• Non-healing wound, ulcer, or bone fracture
• Clinically significant cardiac arrhythmias
• Untreated hypothyroidism
• Uncontrolled systemic infection
• Symptomatic congestive heart failure or unstable angina pectoris within 3 months
prior study drug
• Myocardial infarction, stroke, transient ischemic attack within 6 months
• Known active malignancy (other than glioma) except non-melanoma skin cancer or
carcinoma in-situ in the cervix
6. The subject has inherited bleeding diathesis or coagulopathy with the risk of bleeding
7. The subject has received any of the following prior anticancer therapy:
• Non-standard radiation therapy such as brachytherapy, systemic radioisotope
therapy, or intra-operative radiotherapy (IORT) to the target site.
• Radiation therapy within 12 weeks of screening
• Systemic therapy (including investigational agents and small-molecule kinase
inhibitors) or non-cytotoxic hormonal therapy (eg, tamoxifen) within 14 days or 5
half-lives, whichever is shorter, prior first dose of study drug
• Biologic agents (antibodies, immune modulators, vaccines, cytokines) within 21
days prior to first dose of study drug
• Nitrosoureas or mitomycin C within 42 days, or metronomic/protracted low-dose
chemotherapy within 14 days, or other cytotoxic chemotherapy within 28 days,
prior to first dose of study drug
• Prior treatment with carmustine wafers
• Patients who are currently receiving any other investigational agents and/or who
have received an investigational agent in the prior 28 days
8. Multifocal progression or involvement of the leptomeninges
9. Psychiatric illness/social situations that would limit compliance with the study
requirements
10. Infratentorial disease
Safeguarding the Brain of Our Smallest Infants Phase III (SafeBoosC)
the SafeBoosC-III trial investigates the benefit and harms of treatment based on
near-infrared spectroscopy monitoring compared with treatment as usual. The hypothesis is
that treatment based on near-infrared spectroscopy monitoring for extremely preterm infants
during the first 72 hours of life will result in a reduction in severe brain injury or death
at 36 weeks postmenstrual age.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Lina Chalak
35027
All
up to 6 Hours old
N/A
This study is NOT accepting healthy volunteers
NCT03770741
STU-2019-1707
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:
• Infants born with postmenstrual age less than 28 weeks
• Signed informed consent, unless the NICU has chosen to use 'opt-out' or deferred
consent as consent method.
Exclusion Criteria:
• Missing written parental informed consent (if the 'opt-out' method is used for
consent, lack of a record that the clinical staff have explained the trial and the
'opt-out' consent process to parents and/or a record in the infant's clinical file of
parents' decision to opt-out, are exclusion criteria)
• Decision not to conduct full life support
• No possibility to place cerebral NIRS oximeter within six hours after birth
Other: Modify cardio-respiratory support to avoid cerebral hypoxia, Other: Treatment as usual
Phase I/II Study of MEK162 for Children With Ras/Raf Pathway Activated Tumors
The main purpose of phase I studies in general is to determine the best dose ("maximum
tolerated dose") of a drug, and to find out the most common side effects. The main purpose
of the phase I component of this study specifically is to determine the best dose of the
experimental drug MEK162 and to find out whether the drug is safe in children and
adolescents with tumors that have grown or come back despite standard therapy.
Another purpose of this study is to measure the concentration of drug in the blood to help
understand how much drug gets into the body and how quickly the drug is removed from the
body. Another purpose of this study is to determine whether MEK162 turns off the Ras/Raf/MAP
pathway as expected by measuring this pathway in blood cells. Finally, in this study, the
investigators hope to start finding out whether or not MEK162 causes different types of
tumors in children to shrink or stop growing.
The main purpose of the phase II component of the study is to determine whether MEK162
causes specific types of tumors in children and adolescents to shrink or stop growing. These
specific types of tumors include low-grade gliomas, tumors in patients with a genetic
condition called neurofibromatosis type 1, and other tumors thought to be caused by abnormal
activation of the Ras/Raf/MEK molecular pathway.
Another purpose of this study is for researchers to learn whether specific abnormalities in
the DNA of tumors can help predict whether tumors will respond to MEK162.
• Patients with recurrent, refractory, or progressive non-hematologic malignancies (CNS
or solid tumors) characterized by known or presumed Ras-Raf pathway activation will
be eligible.
• Age >1 year and <18 years.
• Karnofsky Performance Scale (patients > 12 years old) or Lansky Play •Performance
Scale (patients ≤ 12 years old) > 60.
• Normal organ and marrow function as defined below:
• Absolute neutrophil count > 1,000/mcL
• Platelets > 75,000/mcL and > 7 days since last platelet transfusion. Hemoglobin
> 9gm/dL and > 7 days since last red blood cell transfusion
• Not refractory to red cell or platelet transfusions
• Hepatic: Total bilirubin 1.5 times the upper limit of normal; SGPT (ALT) and
SGOT (AST) < 3 times the institutional upper limit of normal.
• Renal: Serum creatinine < 1.5 times the upper limit of institutional normal for
age or GFR > 70 ml/min/1.73m2.
• QTc interval < 450ms.
• Left ventricular ejection fraction (LVEF) > 50%.
• Female patients of childbearing potential must have negative serum or urine pregnancy
test within 72 hours of the first dose of MEK162. Patient must not be pregnant or
breast-feeding. Patients of childbearing or child-fathering potential must be willing
to use a medically acceptable form of birth control, which includes abstinence, while
being treated on this study.
• Patient must be able to take oral/enteral medication.
• Patient, parent, or legal guardian must be able to understand and willing to provide
informed consent.
• Patients must have recovered from the effects of prior therapy.
Exclusion Criteria:
• Patients must not have any significant medical illnesses that in the investigator's
opinion cannot be adequately controlled with appropriate therapy or would compromise
the patient's ability to tolerate this therapy.
• History of Gilbert's syndrome.
• Patients receiving any other anticancer or experimental drug therapy.
• Use of hematopoietic growth factors within 2 weeks prior to initiation of therapy.
• Any other investigational agents within 2 weeks or ≤ 5 x t1/2 (whichever is longer)
before start of study therapy
• Patients who have undergone surgery ≤ 3 weeks or who have not recovered from side
effects of this procedure prior to receiving study drug.
• Uncontrolled intercurrent illness including, but not limited to ongoing or active
infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac
arrhythmia, impaired gastrointestinal function, or psychiatric illness/social
situations that would limit compliance with study requirements.
• History or current evidence of retinal vein occlusion (RVO) or predisposing factors
to RVO (e.g. uncontrolled glaucoma or ocular hypertension, history of hyperviscosity
or hypercoagulability syndromes)
• History of retinal degenerative disease
• Prior therapy with a MEK inhibitor.
• Impairment of gastrointestinal function (e.g., active ulcerative disease,
uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome).
• Patients who have a neuromuscular disorder that is associated with elevated CK (e.g.,
inflammatory myopathies, muscular dystrophy, amyotrophic lateral sclerosis, spinal
muscular atrophy).
Drug: MEK162
Low-Grade Gliomas, Malignant Neoplasms, Brain, Soft Tissue Neoplasms, Brain and Nervous System
Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
This phase II trial studies how well trametinib works in treating patients with juvenile
myelomonocytic leukemia that has come back (relapsed) or does not respond to treatment
(refractory). Trametinib may stop the growth of tumor cells by blocking some of the enzymes
needed for cell growth.
• Patients must have had histologic verification of juvenile myelomonocytic leukemia
(JMML) at original diagnosis and currently have relapsed or refractory disease; the
diagnosis is made based on the following criteria
• JMML category 1 (all of the following): the diagnostic criteria must include all
features in category 1 and EITHER (i) one of the features in category 2 OR (ii)
two features from category 3 to make the diagnosis
• Splenomegaly
• > 1000 (1 x 10^9/uL) circulating monocytes
• < 20% blasts in the bone marrow or peripheral blood
• Absence of the t(9;22) or BCR/ABL fusion gene
• JMML category 2 (at least one of the following if at least two category 3
criteria are not present):
• Somatic mutation in RAS or PTPN11
• Clinical diagnosis of NF1 or NF1 gene mutation
• Homozygous mutation in CBL
• Monosomy 7
• JMML category 3 (at least two of the following if no category 2 criteria are
met):
• Circulating myeloid precursors
• White blood cell count, > 10 000 (10 x 10^9/ uL)
• Increased hemoglobin F for age
• Clonal cytogenetic abnormality
• GM-CSF hypersensitivity
• Patients with refractory or relapsed JMML must have had at least one cycle of
intensive frontline therapy or at least 2 cycles of a deoxyribonucleic acid (DNA)
demethylating agent with persistence of disease, defined by clinical symptoms or the
presence of a clonal abnormality; frontline therapy is defined as one cycle of
intravenous chemotherapy that includes of any of the following agents: fludarabine,
cytarabine, or any anthracycline but specifically excludes oral 6-mercaptopurine;
frontline therapy will also include any conditioning regimen as part of a stem cell
transplant; patients who transform to AML at any point with more than 20% blasts are
not eligible for this trial
• Patients must have a Lansky or Karnofsky performance status score of >= 50,
corresponding to Eastern Cooperative Oncology Group (ECOG) categories 0, 1 or 2; use
Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age;
patients who are unable to walk because of paralysis, but who are up in a wheelchair,
will be considered ambulatory for the purpose of assessing the performance score
• Patients must have fully recovered from the acute toxic effects of all prior
chemotherapy, immunotherapy, or radiotherapy prior to study enrollment
• Myelosuppressive chemotherapy: patients must have completely recovered from all
acute toxic effects of chemotherapy, immunotherapy or radiotherapy prior to study
enrollment; at least 14 days must have elapsed since the completion of cytotoxic
therapy, with the exception of hydroxyurea
• Note: cytoreduction with hydroxyurea can be initiated and continued for up
to 24 hours prior to the start of protocol therapy
• Hematopoietic growth factors: at least 14 days after the last dose of a
long-acting growth factor (e.g., pegfilgrastim) or 7 days for short-acting growth
factor; for agents that have known adverse events occurring beyond 7 days after
administration, this period must be extended beyond the time during which adverse
events are known to occur
• Biologic (anti-neoplastic agent): at least 7 days must have elapsed since
completion of therapy with a biologic agent; for agents that have known adverse
events occurring beyond 7 days after administration, this period prior to
enrollment must be extended beyond the time during which adverse events are known
to occur
• Monoclonal antibodies:
• At least 30 days after the completion of any type of immunotherapy, e.g.
tumor vaccines
• At least 3 half-lives must have elapsed since prior therapy that included a
monoclonal antibody
• Radiotherapy:
• >= 2 weeks must have elapsed since local palliative external radiation
therapy (XRT) (small port)
• >= 6 months must have elapsed if prior craniospinal XRT was received, if >=
50% of the pelvis was irradiated, or if traumatic brain injury (TBI) was
received
• >= 4 weeks must have elapsed if other substantial bone marrow irradiation
was given
• Stem cell transplant or rescue without TBI: no evidence of active graft versus
(vs.) host disease and >= 3 months must have elapsed since transplant; >= 4 weeks
must have elapsed since any donor lymphocyte infusion
• Patients must not be known to be refractory to red blood cell or platelet transfusions
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70
mL/min/1.73 m^2 or a serum creatinine based on age/gender as follows:
• Age: Maximum serum creatinine (mg/dL)
• 1 month to < 6 months: 0.4 (male) 0.4 (female)
• 6 months to < 1 year: 0.5 (male) 0.5 (female)
• 1 to < 2 years: 0.6 (male) 0.6 (female)
• 2 to < 6 years: 0.8 (male) 0.8 (female)
• 6 to < 10 years: 1 (male) 1 (female)
• 10 to < 13 years: 1.2 (male) 1.2 (female)
• 13 to < 16 years: 1.5 (male) 1.4 (female)
• >= 16 years: 1.7 (male) 1.4 (female)
• Total bilirubin =< 1.5 x upper limit of normal (ULN) for age
• Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 3 x
ULN (=< 135 U/L) (for the purpose of this study, the ULN for SGPT is 45 U/L)
• Serum albumin >= 2 g/dL
• Shortening fraction of >= 27% by echocardiogram OR ejection fraction of >= 50% by
multi-gated acquisition (MUGA)
• Corrected QT (by Bazett's formula [QTcB]) interval < 450 msecs
• Patients must be able to swallow tablets or liquid; use of a nasogastric or
gastrostomy (G) tube is also allowed
Exclusion Criteria:
• Patients who are pregnant or breast-feeding are not eligible for this study as there
is yet no available information regarding human fetal or teratogenic toxicities;
negative pregnancy tests must be obtained in girls who are post-menarchal; patients of
reproductive potential may not participate unless they have agreed to use an effective
contraceptive method for the duration of study therapy; women of childbearing
potential should be advised to use effective contraception for 4 months after the last
dose of trametinib; trametinib may also potentially be secreted in milk and therefore
breastfeeding women are excluded; female patients should not breastfeed during
treatment with trametinib, and for 4 months following the last dose; male patients
must use a condom during intercourse and agree not to father a child during therapy
and for 4 months following discontinuation of trametinib to avoid unnecessary exposure
of trametinib to the fetus
• Concomitant Medications
• Corticosteroids: patients requiring corticosteroids who have not been on a stable
or decreasing dose of corticosteroid for the 7 days prior to enrollment are not
eligible; if used to modify immune adverse events related to prior therapy, >= 14
days must have elapsed since last dose of corticosteroid
• Note: hydrocortisone used as a pre-medication to prevent transfusion related
reactions is not considered a concomitant corticosteroid
• Investigational drugs: patients who are currently receiving another
investigational drug are not eligible
• Anti-cancer agents: patients who are currently receiving other anti-cancer agents
are not eligible (except patients receiving hydroxyurea, which may be continued
until 24 hours prior to start of protocol therapy)
• Anti-graft versus host disease (GVHD) or agents to prevent organ rejection
post-transplant: patients who are receiving cyclosporine, tacrolimus or other
agents to prevent either graft-versus-host disease post bone marrow transplant or
organ rejection post-transplant are not eligible for this trial
• Cardiac medications: any medications for treatment of left ventricular systolic
dysfunction
• Patients who have an uncontrolled infection are not eligible
• Patients who in the opinion of the investigator may not be able to comply with the
safety monitoring requirements of the study are not eligible
• Patients with a history of hepatic sinusoid obstructive syndrome (veno-occlusive
disease) within the prior 3 months are not eligible
• Patients with a history of or current evidence/risk of retinal vein occlusion (RVO) or
central serous retinopathy (CSR) are not eligible
• Patients with a history of RVO or CSR, or predisposing factors to RVO or CSR (e.g.,
uncontrolled glaucoma or ocular hypertension
• Patients with uncontrolled systemic disease(s) such as hypertension or diabetes
mellitus are not eligible; blood pressure must be =< the 95th percentile for age,
height, and gender
• Patients with a history of allergic reaction attributed to compounds of similar
chemical or biologic composition to the MEK inhibitor, trametinib are not eligible
• Patients with a clinical diagnosis of Noonan syndrome are not eligible; Note: patients
with Casitas B-lineage lymphoma (CBL) syndrome, also known as Noonan-like syndrome,
are eligible to enroll
Drug: Trametinib
Juvenile Myelomonocytic Leukemia, Neurofibromatosis Type 1
Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 (SPR1NT)
To evaluate the safety and efficacy of intravenous onasemnogene abeparvovec-xioi in
pre-symptomatic patients with SMA and 2 or 3 copies SMN2
Call 214-648-5005 studyfinder@utsouthwestern.edu
Susan Iannaccone
13463
All
up to 42 Days old
Phase 3
This study is NOT accepting healthy volunteers
NCT03505099
STU 022018-080
Show full eligibility criteria
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Inclusion Criteria:
• Age ≤6 weeks (≤42 days) at time of dose
• Ability to tolerate thin liquids as demonstrated through a formal bedside swallowing
test
• Compound muscle action potential (CMAP) ≥2mV at Baseline; centralized review of CMAP
data will be conducted
• Gestational age of 35 to 42 weeks
• Patients with pre-symptomatic SMA Type 1 as determined by the following features:
• 2 copies of SMN2 Patients with 2 copies of SMN2 (n ≥12)
• Patients with pre-symptomatic SMA Type 2 as determined by the following features:
• 3 copies of SMN2
Exclusion Criteria:
• Weight at screening visit <2 kg
• Hypoxemia (oxygen saturation <96% awake or asleep without any supplemental oxygen or
respiratory support) at the screening visit or for altitudes >1000 m, oxygen
saturation <92% awake or asleep without any supplemental oxygen or respiratory support
at the screening visit
• Any clinical signs or symptoms at screening or immediately prior to dosing that are,
in the opinion of the Investigator, strongly suggestive of SMA
• Tracheostomy or current prophylactic use or requirement of noninvasive ventilatory
support at any time and for any duration prior to screening or during the screening
period
• Patients with signs of aspiration/inability to tolerate nonthickened liquids based on
a formal swallowing test performed as part of screening or patients receiving any
non-oral feeding method
• Clinically significant abnormalities in hematology or clinical chemistry parameters as
determined by investigator or medical monitor
• Treatment with an investigational or commercial product, including nusinersen, given
for the treatment of SMA. This includes any history of gene therapy, prior antisense
oligonucleotide treatment, or cell transplantation.
• Patients whose weight-for-age is below the third percentile based on World Health
Organization (WHO) Child Growth Standards
• Biological mother with active viral infection as determined by screening laboratory
samples (includes human immunodeficiency virus [HIV] or positive serology for
hepatitis B or C)
• Biological mothers with clinical suspicion of Zika virus that meet Centers for
Disease Control and Prevention (CDC) Zika virus epidemiological criteria including
history of residence in or travel to a geographic region with active Zika transmission
at the time of travel will be tested for Zika virus RNA. Positive results warrant
confirmed negative Zika virus RNA testing in the patient prior to enrollment.
• Serious nonrespiratory tract illness requiring systemic treatment and/or
hospitalization within 2 Weeks prior to screening
• Upper or lower respiratory infection requiring medical attention, medical
intervention, or increase in supportive care of any manner within 4 Weeks prior to
dosing
• Severe nonpulmonary/respiratory tract infection within 4 Weeks before administration
of gene replacement therapy or concomitant illness that, in the opinion of the
Investigator or Sponsor medical monitor, creates unnecessary risks for gene
replacement therapy such as:
• Major renal or hepatic impairment
• Known seizure disorder
• Diabetes mellitus
• Idiopathic hypocalciuria
• Symptomatic cardiomyopathy
• Known allergy or hypersensitivity to prednisolone or other glucocorticosteroids or
their excipients
• Previous, planned or expected major surgical procedure including scoliosis repair
surgery/procedure during the study assessment period
• Concomitant use of any of the following: drugs for treatment of myopathy or
neuropathy, agents used to treat diabetes mellitus, or ongoing immunosuppressive
therapy, plasmapheresis, immunomodulators such as adalimumab, immunosuppressive
therapy within 4 Weeks prior to gene replacement therapy
• AntiAAV9 antibody titer >1:50 as determined by Enzyme-linked Immunosorbent Assay
(ELISA) binding immunoassay
• Should a potential patient demonstrate AntiAAV9 antibody titer >1:50, he or she may
receive retesting inside the 30-Day screening period and will be eligible to
participate if the AntiAAV9 antibody titer upon retesting is ≤1:50, provided the <6
Week age requirement at the time of dosing is still met
• Biological mother involved with the care of the child refuses anti-AAV9 antibody
testing prior to dosing
1. Age ≥ 18 years.
2. ECOG Performance Score of 2 or better/Karnofsky Performance score of 50-60 or better.
3. Biopsy-proven non-hematopoietic malignancy, except for germ cell cancer. Small cell
lung carcinoma is eligible for this study
4. Six or more metastases on diagnostic or treatment planning imaging, which include
either CT Brain (with contrast) or MR Brain (with or without contrast) imaging.
5. Largest tumor <4 cm
6. No prior SRS to the lesions which will be treated on protocol.
7. Women of child-bearing potential and men must agree to use adequate contraception
(hormonal or barrier method of birth control; abstinence) prior to study entry, for
the duration of study participation, and for 90 days following completion of therapy.
Should a woman become pregnant or suspect she is pregnant while participating in this
study, she should inform her treating physician immediately.
8. A female of child-bearing potential is any woman (regardless of sexual orientation,
marital status, having undergone a tubal ligation, or remaining celibate by choice) who
meets the following criteria:
• Has not undergone a hysterectomy or bilateral oophorectomy; or
• Has not been naturally postmenopausal for at least 12 consecutive months (i.e., has
had menses at any time in the preceding 12 consecutive months).
9 Ability to understand and the willingness to sign a written informed consent.
Exclusion Criteria:
1. Prior whole brain radiotherapy
2. Patients with leptomeningeal metastasis. (NOTE: For the purposes of exclusion, LMD is
a clinical diagnosis, defined as positive CSF cytology and/or equivocal radiologic or
clinical evidence of leptomeningeal involvement. Patients with leptomeningeal symptoms
in the setting of leptomeningeal enhancement by imaging (MRI) would be considered to
have LMD even in the absence of positive CSF cytology, unless a parenchymal lesion can
adequately explain the neurologic symptoms and/or signs. In contrast, an asymptomatic
or minimally symptomatic patient with mild or nonspecific leptomeningeal enhancement
(MRI) would not be considered to have LMD. In that patient, CSF sampling is not
required to formally exclude LMD, but can be performed at the investigator's
discretion based on level of clinical suspicion.)
3. Patients with life expectancy < 4 months
4. Psychiatric illness/social situations that, in the opinion of the investigator, would
limit compliance with study requirements.
5. Subjects must not be pregnant or nursing due to the potential for congenital
abnormalities and the potential of this regimen to harm nursing infants
A Study to Investigate the Efficacy and Safety of ZX008 (Fenfluramine Hydrochloride) as an Adjunctive Therapy in Children and Adults With Lennox-Gastaut Syndrome
This is a two-part, multicenter, double-blind, parallel-group, placebo controlled study to
evaluate the effect of ZX008 when used as adjunctive therapy for the treatment of
uncontrolled seizures in children and adults with Lennox-Gastaut syndrome (LGS).
Call 214-648-5005 studyfinder@utsouthwestern.edu
Deepa Sirsi
103210
All
2 Years to 35 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03355209
STU 072018-033
Show full eligibility criteria
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Key
Inclusion Criteria:
• Male or non-pregnant, non-lactating female, age 2 to 35 years, inclusive as of the day
of the Screening Visit.
• Clinical diagnosis of Lennox-Gastaut syndrome, where seizures that result in drops are
not completely controlled by current antiepileptic treatments.
• Onset of seizures at 11 years of age or younger.
• Abnormal cognitive development.
• Must be receiving at least 1 concomitant AED and up to 4 concomitant anti-epileptic
treatments.
Key
Exclusion Criteria:
• Etiology of seizures is a degenerative neurological disease.
• History of hemiclonic seizures in the first year of life.
• Subject only has drop seizures in clusters, where individual seizures cannot be
counted reliably.
• Pulmonary arterial hypertension.
• Current or past history of cardiovascular or cerebrovascular disease, such as cardiac
valvulopathy, myocardial infarction or stroke.
• Receiving concomitant therapy with: centrally-acting anorectic agents;
monoamineoxidase inhibitors; any centrally-acting compound with clinically appreciable
amount of serotonin agonist or antagonist properties, including serotonin reuptake
inhibition; atomoxetine, or other centrally-acting noradrenergic agonist;
cyproheptadine.
• Taking felbamate for less than 1 year prior to screening and/or does not have stable
liver function and hematology laboratory tests, and/or the dose has not been stable
for at least 60 days prior to the Screening Visit.
• Currently receiving an investigational product.
• Institutionalized in a general nursing home (ie, in a facility that does not
specialize in epilepsy care).
• A clinically significant condition, or has had clinically relevant symptoms or a
clinically significant illness in the 4 weeks prior to the Screening Visit, other than
epilepsy, that would negatively impact study participation, collection of study data,
or pose a risk to the subject.
Drug: ZX008 0.2 or 0.8 mg/kg/day, Drug: Matching Placebo
Veliparib, Radiation Therapy, and Temozolomide in Treating Patients With Newly Diagnosed Malignant Glioma Without H3 K27M or BRAFV600 Mutations
This phase II trial studies how well veliparib, radiation therapy, and temozolomide work in
treating patients with newly diagnosed malignant glioma without H3 K27M or BRAFV600
mutations. Poly adenosine diphosphate (ADP) ribose polymerases (PARPs) are proteins that help
repair DNA mutations. PARP inhibitors, such as veliparib, can keep PARP from working, so
tumor cells can't repair themselves, and they may stop growing. Radiation therapy uses high
energy x-rays to kill tumor cells and shrink tumors. Drugs used in chemotherapy, such as
temozolomide, 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. Giving veliparib,
radiation therapy, and temozolomide may work better in treating patients with newly diagnosed
malignant glioma without H3 K27M or BRAFV600 mutations compared to radiation therapy and
temozolomide alone.
• Stratum 1 (IDH wild-type): Patients must be >= 3 years of age and =< 21 years of age
at the time of enrollment
• Stratum 2 (IDH mutant): Patients must be >= 3 years of age and =< 25 years of age at
the time of enrollment
• Patients must have eligibility confirmed by rapid central pathology and central
molecular screening reviews performed on APEC14B1:
• Newly-diagnosed high-grade glioma such as anaplastic astrocytoma or glioblastoma
• Negative results for H3 K27M by immunohistochemistry (IHC)
• Negative results for BRAFV600 mutation by next-generation sequencing (NGS)
• Patients must have histological verification of diagnosis. Patients with M+ disease
(defined as evidence of neuraxis dissemination) are not eligible. Cerebrospinal fluid
(CSF) cytology is not required but may be obtained if clinically indicated prior to
study enrollment. If cytology is positive, the patient would be considered to have
metastatic disease and would, therefore, be ineligible
• Pre-operative and post-operative brain magnetic resonance imaging (MRI) with and
without contrast must be obtained. The requirement for a post-operative MRI is waived
for patients who undergo biopsy only. A spine MRI is not required, but may be obtained
if clinically indicated. If the spine MRI is positive, the patient would be considered
to have M+ disease (defined as neuraxis dissemination) and would be ineligible
• 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
• Peripheral absolute neutrophil count (ANC) >= 1,000/uL (within 7 days prior to
enrollment)
• Platelet count >= 100,000/uL (transfusion independent) (within 7 days prior to
enrollment)
• Hemoglobin >= 8.0 gm/dL (can be transfused) (within 7 days prior to enrollment)
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70
mL/min/1.73 m^2 OR a serum creatinine based on age/gender as follows (within 7 days
prior to enrollment):
• 3 to < 6 years: 0.8 (male and female) maximum serum creatinine (mg/dL)
• 6 to < 10 years: 1 (male and female) maximum serum creatinine (mg/dL)
• 10 to < 13 years: 1.2 (male and female) maximum serum creatinine (mg/dL)
• 13 to < 16 years: 1.5 (male), 1.4 (female) maximum serum creatinine (mg/dL)
• >= 16 years: 1.7 (male), 1.4 (female) maximum serum creatinine (mg/dL)
• Total bilirubin =< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to
enrollment)
• Serum glutamic-oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST]) or
serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) < 2.5 x
upper limit of normal (ULN) for age (within 7 days prior to enrollment)
• Patients with seizure disorder may be enrolled if seizures are well-controlled (i.e.,
patients must not have required rescue medications for uncontrolled seizures within 14
days prior to enrollment)
• Patients must be enrolled and protocol therapy must be projected to begin no later
than 31 days after definitive diagnostic surgery (Day 0)
• All patients and/or their parents or legal guardians must sign a written informed
consent
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute
(NCI) requirements for human studies must be met
Exclusion Criteria:
• Patients with the following histologies:
• Diffuse astrocytoma (grade 2)
• Oligodendrogliomas (any grade)
• Pleomorphic xanthoastrocytoma (PXA, any grade)
• Patients with primary tumor location of brainstem or spinal cord
• Patients with M+ disease (defined as neuraxis dissemination either by imaging or by
cytology)
• Patients with treatment-related acute myeloid leukemia (AML) (t-AML)/myelodysplastic
syndrome (MDS) or with features suggestive of AML/MDS
• Prior allogenic bone marrow transplant or double umbilical cord blood transplantation
• Patients must not have received any prior tumor-directed therapy including radiation
therapy, chemotherapy (tumor-directed therapy), molecularly targeted agents, or
immunotherapy for the treatment of HGG other than surgical intervention and/or
corticosteroids
• Lumbar CSF cytology is not required, but may be performed if clinically indicated
prior to study enrollment. If lumbar CSF cytology is positive, the patient is
considered to have M+ disease and is ineligible
• Note: False positive cytology can occur within 10 days of surgery
• Patients with gliomatosis cerebri type 1 or 2
• Patients who are not able to receive protocol specified radiation therapy
• Patients must not be currently receiving other anti-cancer agents
• Patients with known constitutional mismatch repair deficiency syndrome
(CMMR-D)/biallelic mismatch repair deficiency (bMMRD)
• Female patients who are pregnant are ineligible due to risks of fetal and teratogenic
adverse events as seen in animal/human studies
• Lactating females are not eligible unless they have agreed not to breastfeed their
infants
• Female patients of childbearing potential are not eligible unless a negative pregnancy
test result has been obtained
• Sexually active patients of reproductive potential are not eligible unless they have
agreed to use an effective contraceptive method for the duration of their study
participation and for 6 months after the last dose of protocol-specified chemotherapy
Sleep for Stroke Management and Recovery Trial (Sleep SMART)
The purpose of this study is to determine whether treatment of obstructive sleep apnea (OSA)
with positive airway pressure starting shortly after acute ischemic stroke or high risk TIA
(1) reduces recurrent stroke, acute coronary syndrome, and all-cause mortality 6 months after
the event, and (2) improves stroke outcomes at 3 months in patients who experienced an
ischemic stroke.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Mehari Gebreyohanns
141046
All
18 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT03812653
STU-2019-0861
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Inclusion Criteria:
1. TIA with ABCD2 ≥4 or ischemic stroke, within the prior 14 days.
Exclusion Criteria:
1. pre-event inability to perform all of own basic ADLs
2. unable to obtain informed consent from subject or legally authorized representative
3. incarcerated
4. known pregnancy
5. current mechanical ventilation (can enroll later if this resolves) or tracheostomy
6. current use of positive airway pressure, or use within one month prior to stroke
7. anatomical or dermatologic anomaly that makes use of CPAP interface unfeasible
8. severe bullous lung disease
9. history of prior spontaneous pneumothorax or current pneumothorax
10. hypotension requiring current treatment with pressors (can enroll later if this
resolves)
11. other specific medical circumstances that conceivably, in the opinion of the site PI,
could render the patient at risk of harm from use of CPAP
12. massive epistaxis or previous history of massive epistaxis
13. cranial surgery or head trauma within the past 6 months, with known or possible CSF
leak or pneumocephalus
14. recent hemicraniectomy or suboccipital craniectomy (i.e. those whose bone has not yet
been replaced), or any other recent bone removal procedure for relief of intracranial
pressure
15. current receipt of oxygen supplementation >4 liters per minute
16. current contact, droplet, respiratory/airborne precautions
A Study to Evaluate the Safety and Efficacy of IPX203 in Parkinson's Disease Patients With Motor Fluctuations
To evaluate the safety and efficacy of IPX203 (carbidopa and levodopa) extended-release
capsules (IPX203 ER CD-LD) in comparison to immediate release (IR) CD-LD in the treatment of
CD-LD-experienced subjects with Parkinson's disease (PD) who have motor fluctuations.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Padraig O'Suilleabhain
35895
All
40 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT03670953
STU-2018-0410
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Inclusion Criteria:
• Male or female subjects diagnosed at age ≥ 40 years with PD, consistent with the
United Kingdom Parkinson's Disease Society Brain Bank Diagnostic Criteria and who are
being treated with stable regimens of CD-LD but experiencing motor fluctuations.
• Able to provide written informed consent prior to the conduct of any study-specific
procedures.
• Female subjects of childbearing potential must have a negative urine pregnancy test at
Screening Visit.
• Negative urine screen for drugs of abuse and negative alcohol breath test at
Screening.
• Hoehn and Yahr Stages 1, 2, 3, or 4 in the "On" state (part of Movement Disorders
Society version of the Unified Parkinson's Disease Rating Scale [MDS-UPDRS] Part III)
• Agrees to use a medically acceptable method of contraception throughout the study and
for 6 weeks after completing the study. Medically acceptable methods of contraception
that may be used by the subject and/or partner include but are not limited to:
abstinence, oral contraception, NuvaRing or transdermal systems, diaphragm with
vaginal spermicide, intrauterine device, condom and partner using vaginal spermicide,
surgical sterilization (6 months), progestin implant or injection, or postmenopausal
female (no menstrual period for ˃ 2 years) or vasectomy (˃ 6 months).
• Montreal Cognitive Assessment (MoCA) score ≥ 24 at Screening Visit in "On" state.
• Able to differentiate "On" state from "Off" state as determined by at least 75%
concordance with a trained rater in "On/Off" ratings for 8 ratings over a 4-hour
training period. The concordance must include at least 1 "On" and 1 "Off" rating and
must be achieved within two 4-hour training sessions.
• Able and willing to comply with the protocol, including completion of diaries and
availability for all study visits.
• Responsive to CD-LD therapy and currently being treated on a stable regimen with CD-LD
for at least 4 weeks prior to Visit 1.
• At Screening, the subject has predictable "Off" periods.
Exclusion Criteria:
• Received any investigational medications within 30 days or 5 times the half-life,
whichever is longer, prior to Visit 1.
• Female subjects who are currently breastfeeding or lactating.
• Had prior neurosurgical treatment for PD or if such procedure is planned or
anticipated during the study period.
• Allergic to any excipient in the study drugs.
• History of medical conditions or of a prior surgical procedure that would interfere
with LD absorption, such as gastrectomy, proximal small-bowel resection, or bariatric
surgery.
• History of upper gastrointestinal hemorrhage in patients with peptic ulcer disease
within the past 5 years.
• History of glaucoma with intraocular pressures that are elevated despite appropriate
medical management.
• History of seizure or epilepsy and experienced at least 1 seizure during the past 12
months or has not been compliant with medically recommended therapy or visits.
• History of myocardial infarction with residual atrial, nodal, or ventricular
arrhythmias that are not controlled with medical and/or surgical interventions. A
recent (≤ 12 months) history of myocardial infarction with secondary arrhythmias is
exclusionary regardless of the therapeutic control.
• History of neuroleptic malignant syndrome or of nontraumatic rhabdomyolysis.
• Liver enzyme values ≥ 2.5 times the upper limit of normal; or history of severe
hepatic impairment.
• Serum creatinine level ≥ 1.75 times the upper limit of normal; or requires dialysis at
the time of Screening.
• Subject with a history of malignant melanoma or with a suspicious undiagnosed skin
lesion which in the opinion of the investigator could be melanoma.
• History of drug or alcohol abuse within the 12 months prior to Screening.
• Received within 4 weeks of Screening or planning to take during participation in the
clinical study:
• Any doses of a CR CD-LD apart from a single daily bedtime dose, any doses of Rytary,
additional CD (eg, Lodosyn) or benserazide (eg, Serazide), or catechol-O-methyl
transferase inhibitors (entacapone or tolcapone) or medications containing these
inhibitors (Stalevo),
• Nonselective monoamine oxidase inhibitors (MAOI), apomorphine, or antidopaminergic
agents, including antiemetics.
• Employees or family members of the investigator, study site, or sponsor.
• Subjects who have previously participated in an IPX203 study.
• Subjects who, in the opinion of the clinical investigator, should not participate in
the study.
• Based on clinical assessment, subject does not adequately comprehend the terminology
needed to complete the PD diary.
Drug: IR CD-LD, Drug: IPX203 ER CD-LD, Other: IPX203 placebo, Other: IR CD-LD placebo
Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy (ATTRIBUTE-CM)
Phase 3 efficacy and safety study to evaluate AG10 800 mg compared to placebo in subjects
with symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
Call 214-648-5005 studyfinder@utsouthwestern.edu
Justin Grodin
74652
All
18 Years to 90 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03860935
STU-2018-0350
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Inclusion Criteria:
• Have an established diagnosis of ATTR-CM with either wild-type TTR or variant TTR
genotype
• Have a history of heart failure evidenced by at least one prior hospitalization for
heart failure or clinical evidence of heart failure without prior heart failure
hospitalization manifested by signs or symptoms of volume overload or elevated
intracardiac pressures or heart failure symptoms that required or require ongoing
treatment with a diuretic.
• New York Heart Association (NYHA) Class I-III symptoms due to ATTR cardiomyopathy.
• On stable doses of cardiovascular medical therapy
• Completed ≥150 m on the 6MWT on 2 tests prior to randomization
• Biomarkers of myocardial wall stress, NT-proBNP level ≥300 pg/mL
• Have left ventricular wall (interventricular septum or left ventricular posterior
wall) thickness ≥12 mm
Exclusion Criteria:
• Had acute myocardial infarction, acute coronary syndrome or coronary
revascularization, or experienced stroke or transient ischemic attack within 90 days
prior to screening
• Has hemodynamic instability
• Likely to undergo heart transplantation within a year of screening
• Confirmed diagnosis of primary (light chain) amyloidosis
• Biomarkers of myocardial wall stress, NT-proBNP level ≥8500 pg/mL at screening
• Measure of kidney function, eGFR by MDRD formula <15 mL/min/1.73 m2
• Current treatment with marketed drug products and other investigational agents for the
treatment of ATTR-CM
• Current treatment with calcium channel blockers with conduction system effects (e.g.
verapamil, diltiazem). The use of dihydropyridine calcium channel blockers is allowed.
The use of digitalis will only be allowed if required for management of atrial
fibrillation with rapid ventricular response