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Showing 1–24 of 625 matching trials from the live ClinicalTrials.gov search.
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No Longer Available No phase listed Expanded access

Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

NCT01461304
View directory record Official record
Conditions
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency, Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency, Glycogen Storage Disorders, Pyruvate Carboxylase Deficiency Disease, ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of, Barth Syndrome
Interventions
triheptanoin
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
1 Month and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 9, 2021 · Synced Jul 22, 2026, 3:45 AM EDT
Not listed No phase listed Observational

Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping

NCT01772043
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
4 Years and older · Male only
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
8
States / cities
Sacramento, California • Stanford, California • Washington D.C., District of Columbia + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 27, 2015 · Synced Jul 22, 2026, 3:45 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy

NCT02439216
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Edasalonexent, Placebo
Drug
Lead sponsor
Catabasis Pharmaceuticals
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
5
States / cities
Los Angeles, California • Gainesville, Florida • Orlando, Florida + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2022 · Synced Jul 22, 2026, 3:45 AM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Derivation of Human Induced Pluripotent Stem (iPS) Cells to Heritable Cardiac Arrhythmias

NCT02413450
View directory record Official record
Conditions
Inherited Cardiac Arrythmias, Long QT Syndrome (LQTS), Brugada Syndrome (BrS), Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT), Early Repolarization Syndrome (ERS), Arrhythmogenic Cardiomyopathy (AC, ARVD/C), Hypertrophic Cardiomyopathy (HCM), Dilated Cardiomyopathy (DCM), Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Normal Control Subjects
Interventions
Not listed
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 85 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2031
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 15, 2026 · Synced Jul 22, 2026, 3:45 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Phenotype, Genotype and Biomarkers 2

NCT04875416
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Hereditary Spastic Paraplegia, Primary Lateral Sclerosis, Progressive Muscular Atrophy, Frontotemporal Dementia
Interventions
Not listed
Lead sponsor
University of Miami
Other
Eligibility
7 Years and older
Enrollment
217 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2031
U.S. locations
3
States / cities
Miami, Florida • Kansas City, Kansas • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 14, 2026 · Synced Jul 22, 2026, 3:45 AM EDT
Active, not recruiting Phase 3 Interventional Results available

Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

NCT04281485
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
PF-06939926, Placebo
Genetic · Other
Lead sponsor
Pfizer
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
114 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2039
U.S. locations
17
States / cities
Little Rock, Arkansas • Los Angeles, California • Gainesville, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 1, 2026 · Synced Jul 22, 2026, 3:45 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Natural History Study - Mitochondrial Disease

NCT01532791
View directory record Official record
Conditions
MELAS or m.3243 A>G Mitochondrial DNA Mutation Carrier
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
4 Years and older
Enrollment
300 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced Jul 22, 2026, 3:45 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1

NCT06844214
View directory record Official record
Conditions
Myotonic Dystrophy
Interventions
SAR446268
Biological
Lead sponsor
Sanofi
Industry
Eligibility
10 Years to 55 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
4
States / cities
Gainesville, Florida • Tampa, Florida • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated May 31, 2026 · Synced Jul 22, 2026, 3:45 AM EDT
Terminated No phase listed Observational

A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.

NCT01753804
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Observational study
Other
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
3 Years to 18 Years · Male only
Enrollment
269 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
3
States / cities
Sacramento, California • Cincinnati, Ohio • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 7, 2017 · Synced Jul 22, 2026, 3:45 AM EDT
Completed Phase 2 Interventional Results available

Olaparib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Defects in DNA Damage Repair Genes (A Pediatric MATCH Treatment Trial)

NCT03233204
View directory record Official record
Conditions
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma, Ann Arbor Stage IV Childhood Non-Hodgkin Lymphoma, Low Grade Glioma, Malignant Glioma, Recurrent Childhood Central Nervous System Neoplasm, Recurrent Childhood Ependymoma, Recurrent Childhood Malignant Germ Cell Tumor, Recurrent Childhood Non-Hodgkin Lymphoma, Recurrent Childhood Rhabdomyosarcoma, Recurrent Childhood Soft Tissue Sarcoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Glioma, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Osteosarcoma, Refractory Childhood Malignant Germ Cell Tumor, Refractory Ependymoma, Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Refractory Glioma, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Primary Central Nervous System Neoplasm, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Rhabdoid Tumor, Wilms Tumor
Interventions
Olaparib
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Months to 21 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
111
States / cities
Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 88 more
Source: ClinicalTrials.gov public record
Updated Dec 10, 2024 · Synced Jul 22, 2026, 3:45 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Evaluate Safety and Biological Activity of ATYR1940 in Participants With Early Onset Facioscapulohumeral Muscular Dystrophy

NCT02603562
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy (FSHD)
Interventions
ATYR1940
Biological
Lead sponsor
aTyr Pharma, Inc.
Industry
Eligibility
16 Years to 25 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016
U.S. locations
4
States / cities
Stanford, California • Iowa City, Iowa • Columbus, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 18, 2023 · Synced Jul 22, 2026, 3:45 AM EDT
Completed Phase 3 Interventional

High-dose Prednisone in Duchenne Muscular Dystrophy

NCT00110669
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Prednisone
Drug
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
4 Years to 10 Years · Male only
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2008
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Oct 26, 2011 · Synced Jul 22, 2026, 3:45 AM EDT
Terminated Phase 3 Interventional Results available

A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)

NCT03783923
View directory record Official record
Conditions
Limb-Girdle Muscular Dystrophy
Interventions
Deflazacort
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
8
States / cities
Atlanta, Georgia • Iowa City, Iowa • Kansas City, Kansas + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 26, 2022 · Synced Jul 22, 2026, 3:45 AM EDT
Recruiting Phase 1 Interventional

Immunotherapy For Adults With GPC3-Positive Solid Tumors Using IL-15 and IL-21 Armored GPC3-CAR T Cells

NCT06198296
View directory record Official record
Conditions
Hepatoblastoma, Hepatocellular Carcinoma, Wilms Tumor, Malignant Rhabdoid Tumor, Yolk Sac Tumor, Rhabdomyosarcoma, Liposarcoma, Embryonal Sarcoma of Liver
Interventions
21.15.GPC3-CAR T cells
Genetic
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Up to 21 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2043
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Nov 2, 2025 · Synced Jul 22, 2026, 3:45 AM EDT
Recruiting Phase 1 Interventional

Dexamethasone Use in Pediatric Rhabdomyolysis Patients in Addition to Standard Protocols

NCT06429982
View directory record Official record
Conditions
Rhabdomyolysis
Interventions
Dexamethasone, Placebo
Drug
Lead sponsor
Children's National Research Institute
Other
Eligibility
6 Months to 25 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jul 2, 2025 · Synced Jul 22, 2026, 3:45 AM EDT
Recruiting Phase 1Phase 2 Interventional

Study of Onivyde With Talazoparib or Temozolomide in Children With Recurrent Solid Tumors and Ewing Sarcoma

NCT04901702
View directory record Official record
Conditions
Recurrent Solid Tumor, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Solid Neoplasm, Recurrent Neuroblastoma, Recurrent Osteosarcoma, Recurrent Peripheral Primitive Neuroectodermal Tumor, Recurrent Rhabdoid Tumor, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Recurrent Wilms Tumor, Refractory Ewing Sarcoma, Refractory Hepatoblastoma, Refractory Malignant Germ Cell Tumor, Refractory Malignant Solid Neoplasm, Refractory Neuroblastoma, Refractory Osteosarcoma, Refractory Peripheral Primitive Neuroectodermal Tumor, Refractory Rhabdoid Tumor, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma
Interventions
Onivyde, Talazoparib, Temozolomide
Drug
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
12 Months to 30 Years
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
7
States / cities
Palo Alto, California • Aurora, Colorado • Washington D.C., District of Columbia + 4 more
Source: ClinicalTrials.gov public record
Updated May 18, 2026 · Synced Jul 22, 2026, 3:45 AM EDT
Recruiting Phase 2 Interventional

A Study of Cabozantinib as a Maintenance Agent to Prevent Progression or Recurrence in High-Risk Pediatric Solid Tumors

NCT05135975
View directory record Official record
Conditions
Neuroblastoma, Sarcoma
Interventions
Cabozantinib
Drug
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
18 Months to 40 Years
Enrollment
86 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
6
States / cities
Birmingham, Alabama • Aurora, Colorado • Washington D.C., District of Columbia + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 11, 2025 · Synced Jul 22, 2026, 3:45 AM EDT
Completed Phase 1 Interventional

A Study of CD45RA+ Depleted Haploidentical Stem Cell Transplantation in Children With Relapsed or Refractory Solid Tumors and Lymphomas

NCT01625351
View directory record Official record
Conditions
Ewing Sarcoma, Gastrointestinal Tumor, Germ Cell Tumor, Hepatic Tumor, Lymphoma, Wilms Tumor, Rhabdoid Tumor, Clear Cell Carcinoma, Renal Cell Carcinoma, Melanoma, Neuroblastoma, Rhabdomyosarcoma, Non-rhabdomyosarcoma
Interventions
alemtuzumab, fludarabine, sirolimus, Busulfan, melphalan, stem cells, CliniMACS
Drug · Biological · Device
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
2 Years to 21 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2020
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Feb 5, 2026 · Synced Jul 22, 2026, 3:45 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy

NCT06907875
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy
Interventions
EPI-321
Biological
Lead sponsor
Epicrispr Biotechnologies, Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
5
States / cities
Los Angeles, California • Atlanta, Georgia • Baltimore, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 19, 2026 · Synced Jul 22, 2026, 3:45 AM EDT
Completed No phase listed Observational

Sjogren-Larsson Syndrome: Natural History, Clinical Variation and Evaluation of Biochemical Markers

NCT01971957
View directory record Official record
Conditions
Sjogren-Larsson Syndrome (SLS)
Interventions
Not listed
Lead sponsor
University of Nebraska
Other
Eligibility
Not listed
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
2
States / cities
Omaha, Nebraska • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 7, 2024 · Synced Jul 22, 2026, 3:45 AM EDT
Not listed Phase 1 Interventional

Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B

NCT03882437
View directory record Official record
Conditions
Danon Disease
Interventions
RP-A501
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
8 Years and older · Male only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
3
States / cities
La Jolla, California • Aurora, Colorado • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 2, 2023 · Synced Jul 22, 2026, 3:45 AM EDT
Completed Phase 4 Interventional Accepts healthy volunteers

Tadalafil in Becker Muscular Dystrophy

NCT01070511
View directory record Official record
Conditions
Becker Muscular Dystrophy
Interventions
Tadalafil, Placebo
Drug
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
18 Years to 55 Years · Male only
Enrollment
48 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2012
U.S. locations
2
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Aug 19, 2013 · Synced Jul 22, 2026, 3:45 AM EDT
Completed No phase listed Observational

The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parents, Clinician Researchers, and the Industry Sponsor

NCT01182324
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 99 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 4, 2018 · Synced Jul 22, 2026, 3:45 AM EDT
Terminated Phase 4 Interventional Results available

Desipramine in Infantile Neuroaxonal Dystrophy (INAD).

NCT03726996
View directory record Official record
Conditions
Infantile Neuroaxonal Dystrophy
Interventions
Desipramine
Drug
Lead sponsor
Duke University
Other
Eligibility
3 Years to 17 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 13, 2020 · Synced Jul 22, 2026, 3:45 AM EDT