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Showing 121–144 of 237 matching trials from the live ClinicalTrials.gov search.
Conditions
Duchenne Muscular Dystrophy
Interventions
scAAV9.U7.ACCA
Biological
Lead sponsor
Megan Waldrop
Other
Eligibility
6 Months to 13 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Columbus, Ohio
Conditions
Duchenne Muscular Dystrophy
Interventions
wearable technology
Device
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
10 Years and older · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
1
States / cities
Nashville, Tennessee
Conditions
Muscular Dystrophy, Duchenne
Interventions
Eteplirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 13 Years · Male only
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
3
States / cities
Birmingham, Alabama • Gainesville, Florida • Atlanta, Georgia
Conditions
Stress
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years and older
Enrollment
228 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Terminated Phase 1 Interventional Results available

Spironolactone Versus Prednisolone in DMD

NCT03777319
Conditions
Muscular Dystrophy, Duchenne
Interventions
Spironolactone, Prednisolone
Drug
Lead sponsor
Kevin Flanigan
Other
Eligibility
4 Years to 7 Years · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
4
States / cities
Iowa City, Iowa • Columbus, Ohio • Philadelphia, Pennsylvania + 1 more
Recruiting No phase listed Observational Accepts healthy volunteers

Biomarker Development for Muscular Dystrophies

NCT05019625
Conditions
Myotonic Dystrophy, Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
5 Years and older
Enrollment
465 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2028
U.S. locations
5
States / cities
Boston, Massachusetts • Winston-Salem, North Carolina • Pittsburgh, Pennsylvania
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec, placebo
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
126 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
23
States / cities
Little Rock, Arkansas • La Jolla, California • Los Angeles, California + 20 more
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Early Check: Expanded Screening in Newborns

NCT03655223
Conditions
Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy, Hyperinsulinemic Hypoglycemia, Familial 1, Diabetes Mellitus, Adrenoleukodystrophy, Neonatal, Medium-chain Acyl-CoA Dehydrogenase Deficiency, Very Long Chain Acyl Coa Dehydrogenase Deficiency, Beta-ketothiolase Deficiency, Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency, Primary Hyperoxaluria Type 1, Congenital Bile Acid Synthesis Defect Type 2, Pyridoxine-Dependent Epilepsy, Hereditary Fructose Intolerance, Hypophosphatasia, Hyperargininemia, Mucopolysaccharidosis Type 6, Argininosuccinic Aciduria, Citrullinemia, Type I, Wilson Disease, Maple Syrup Urine Disease, Type 1A, Maple Syrup Urine Disease, Type 1B, Biotinidase Deficiency, Neonatal Severe Primary Hyperparathyroidism, Intrinsic Factor Deficiency, Usher Syndrome Type 1D/F Digenic (Diagnosis), Cystic Fibrosis, Stickler Syndrome Type 2, Stickler Syndrome Type 1, Alport Syndrome, Autosomal Recessive, Alport Syndrome, X-Linked, Carbamoyl Phosphate Synthetase I Deficiency Disease, Carnitine Palmitoyl Transferase 1A Deficiency, Carnitine Palmitoyltransferase II Deficiency, Cystinosis, Chronic Granulomatous Disease, Cerebrotendinous Xanthomatoses, Maple Syrup Urine Disease, Type 2, Severe Combined Immunodeficiency Due to DCLRE1C Deficiency, Thyroid Dyshormonogenesis 6, Thyroid Dyshormonogenesis 5, Supravalvar Aortic Stenosis, Factor X Deficiency, Hemophilia A, Hemophilia B, Tyrosinemia, Type I, Fructose 1,6 Bisphosphatase Deficiency, Glycogen Storage Disease Type I, G6PD Deficiency, Glycogen Storage Disease II, Galactokinase Deficiency, Mucopolysaccharidosis Type IV A, Galactosemias, Guanidinoacetate Methyltransferase Deficiency, Agat Deficiency, Glutaryl-CoA Dehydrogenase Deficiency, Gtp Cyclohydrolase I Deficiency, Hyperinsulinism-Hyperammonemia Syndrome, Primary Hyperoxaluria Type 2, 3-Hydroxyacyl-CoA Dehydrogenase Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency, Mitochondrial Trifunctional Protein Deficiency, Sickle Cell Disease, Beta-Thalassemia, Holocarboxylase Synthetase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, Primary Hyperoxaluria Type 3, Hermansky-Pudlak Syndrome 1, Hermansky-Pudlak Syndrome 4, Apparent Mineralocorticoid Excess, HSDB, CBAS1, Mucopolysaccharidosis Type 2, Mucopolysaccharidosis Type 1, Severe Combined Immunodeficiency, X Linked, Severe Combined Immunodeficiency Due to IL-7Ralpha Deficiency, Diabetes Mellitus, Permanent Neonatal, Isovaleric Acidemia, Severe Combined Immunodeficiency T-Cell Negative B-Cell Positive Due to Janus Kinase-3 Deficiency (Disorder), Jervell and Lange-Nielsen Syndrome 2, Hyperinsulinemic Hypoglycemia, Familial, 2, Diabetes Mellitus, Permanent Neonatal, With Neurologic Features, Jervell and Lange-Nielsen Syndrome 1, Lysosomal Acid Lipase Deficiency, CblF, 3-Methylcrotonyl CoA Carboxylase 1 Deficiency, 3-Methylcrotonyl CoA Carboxylase 2 Deficiency, Waardenburg Syndrome Type 2A, Methylmalonic Aciduria cblA Type, Methylmalonic Aciduria cblB Type, Methylmalonic Aciduria and Homocystinuria Type cblC, MAHCD, Methylmalonic Aciduria Due to Methylmalonyl-CoA Mutase Deficiency, Congenital Disorder of Glycosylation Type 1B, Mthfr Deficiency, Methylcobalamin Deficiency Type Cbl G (Disorder), Methylcobalamin Deficiency Type cblE, Usher Syndrome, Type 1B, N-acetylglutamate Synthase Deficiency, Ornithine Transcarbamylase Deficiency, Phenylketonurias, Waardenburg Syndrome Type 1, Congenital Hypothyroidism, Propionic Acidemia, Usher Syndrome, Type 1F, Pancreatic Agenesis 1, Hereditary Hypophosphatemic Rickets, Glycogen Storage Disease IXB, Glycogen Storage Disease IXC, MOWS, Epilepsy, Early-Onset, Vitamin B6-Dependent, Pyridoxal Phosphate-Responsive Seizures, Pituitary Hormone Deficiency, Combined, 1, Ptsd, Dihydropteridine Reductase Deficiency, Severe Combined Immunodeficiency Due to RAG1 Deficiency, Severe Combined Immunodeficiency Due to RAG2 Deficiency, Retinoblastoma, Multiple Endocrine Neoplasia Type 2B, Pseudohypoaldosteronism, Type I, Liddle Syndrome, Biotin-Responsive Basal Ganglia Disease, SCD, DIAR1, GSD1C, Acrodermatitis Enteropathica, Thyroid Dyshormonogenesis 1, Riboflavin Transporter Deficiency, Waardenburg Syndrome, Type 2E, SRD, Congenital Lipoid Adrenal Hyperplasia Due to STAR Deficiency, Barth Syndrome, Adrenocorticotropic Hormone Deficiency, Transcobalamin II Deficiency, Thyroid Dyshormonogenesis 3, Segawa Syndrome, Autosomal Recessive, Autosomal Recessive Nonsyndromic Hearing Loss, Thyroid Dyshormonogenesis 2A, Congenital Isolated Thyroid Stimulating Hormone Deficiency, Hypothyroidism Due to TSH Receptor Mutations, Usher Syndrome Type 1C, Usher Syndrome Type 1G (Diagnosis), Von Willebrand Disease, Type 3, Combined Immunodeficiency Due to ZAP70 Deficiency, Adenine Phosphoribosyltransferase Deficiency, Metachromatic Leukodystrophy, Canavan Disease, Menkes Disease, Carbonic Anhydrase VA Deficiency, Developmental and Epileptic Encephalopathy 2, 17 Alpha-Hydroxylase Deficiency, Smith-Lemli-Opitz Syndrome, Krabbe Disease, Glutathione Synthetase Deficiency, Mucopolysaccharidosis Type 7, Rett Syndrome, Molybdenum Cofactor Deficiency, Type A, Niemann-Pick Disease, Type C1, Niemann-Pick Disease Type C2, Ornithine Aminotransferase Deficiency, 3-Phosphoglycerate Dehydrogenase Deficiency, Leber Congenital Amaurosis 2, Dravet Syndrome, Mucopolysaccharidosis Type 3 A, Ornithine Translocase Deficiency, Carnitine-acylcarnitine Translocase Deficiency, Glucose Transporter Type 1 Deficiency Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type A, Pitt Hopkins Syndrome, Tuberous Sclerosis 1, Tuberous Sclerosis 2, Ataxia With Isolated Vitamin E Deficiency, Angelman Syndrome, Prader-Willi Syndrome, Homocystinuria, Permanent Neonatal Diabetes Mellitus, Transient Neonatal Diabetes Mellitus, Factor VII Deficiency, Glycogen Storage Disease Type IXA1, Glycogen Storage Disease, Type IXA2, Glycogen Storage Disease IC, Glycogen Storage Disease Type IB, Central Hypoventilation Syndrome With or Without Hirschsprung Disease
Interventions
Confirmatory Testing
Diagnostic Test
Lead sponsor
RTI International
Other
Eligibility
1 Day to 31 Days
Enrollment
30,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2026
U.S. locations
1
States / cities
Research Triangle Park, North Carolina
Conditions
Muscular Dystrophy, Duchenne
Interventions
PF-06939926
Genetic
Lead sponsor
Pfizer
Industry
Eligibility
2 Years to 3 Years · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
10
States / cities
Gainesville, Florida • Philadelphia, Pennsylvania • Salt Lake City, Utah
Conditions
Duchenne Muscular Dystrophy
Interventions
(+)- Epicatechin
Drug
Lead sponsor
Craig McDonald, MD
Other
Eligibility
8 Years to 17 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Sacramento, California
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
SAT-3247
Drug
Lead sponsor
Satellos Bioscience, Inc.
Industry
Eligibility
16 Years to 40 Years · Male only
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
3
States / cities
Los Angeles, California • Columbus, Ohio • Memphis, Tennessee
Conditions
Duchenne Muscular Dystrophy
Interventions
RGX-202
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
1 Year and older · Male only
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
19
States / cities
Little Rock, Arkansas • Orange, California • Palo Alto, California + 16 more
Conditions
Constipation, DMD
Interventions
SmartPill ingestion
Device
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
18 Years and older · Male only
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Cincinnati, Ohio
Completed No phase listed Observational Accepts healthy volunteers

UTSW HP [13-C] Pyruvate Injection in HCM

NCT03057002
Conditions
Cardiomyopathy, Hypertrophic, Dilated Cardiomyopathy, Duchenne Muscular Dystrophy, Cardiac Sarcoidosis, Becker Muscular Dystrophy, Heart Failure With Preserved Ejection Fraction, Heart Failure With Reduced Ejection Fraction
Interventions
Hyperpolarized 13C-Pyruvate
Diagnostic Test
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
18 Years to 60 Years
Enrollment
7 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2023
U.S. locations
1
States / cities
Dallas, Texas
Conditions
Duchenne Muscular Dystrophy
Interventions
givinostat, placebo
Drug
Lead sponsor
Italfarmaco
Industry
Eligibility
6 Years to 17 Years · Male only
Enrollment
179 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
13
States / cities
Davis, California • San Diego, California • Aurora, Colorado + 10 more
Conditions
Duchenne Muscular Dystrophy
Interventions
WVE-210201 (suvodirsen), Placebo
Drug
Lead sponsor
Wave Life Sciences USA, Inc.
Industry
Eligibility
5 Years to 12 Years · Male only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
6
States / cities
New Haven, Connecticut • Atlanta, Georgia • Kansas City, Kansas + 3 more
Conditions
Becker Muscular Dystrophy
Interventions
Sevasemten 10 mg, Sevasemten 5 mg, Sevasemten 12.5 mg, Placebo
Drug
Lead sponsor
Edgewise Therapeutics, Inc.
Industry
Eligibility
12 Years to 50 Years · Male only
Enrollment
244 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
26
States / cities
Little Rock, Arkansas • La Jolla, California • Los Angeles, California + 23 more
Conditions
Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases, Neuromuscular Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn, Nervous System Diseases
Interventions
Deramiocel (CAP-1002), Placebo
Biological
Lead sponsor
Capricor Inc.
Industry
Eligibility
10 Years and older · Male only
Enrollment
106 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
20
States / cities
Phoenix, Arizona • Little Rock, Arkansas • La Jolla, California + 17 more
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Shriners Hospitals for Children
Other
Eligibility
4 Years to 21 Years · Male only
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2015
U.S. locations
3
States / cities
Los Angeles, California • Sacramento, California • Portland, Oregon
Conditions
Duchenne Muscular Dystrophy
Interventions
Delandistrogene Moxeparvovec, Standard of Care
Genetic · Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years and older · Male only
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2038
U.S. locations
25
States / cities
Little Rock, Arkansas • Los Angeles, California • Aurora, Colorado + 22 more
Conditions
Duchenne Muscular Dystrophy
Interventions
WVE-N531
Drug
Lead sponsor
Wave Life Sciences USA, Inc.
Industry
Eligibility
4 Years to 18 Years · Male only
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
2
States / cities
Little Rock, Arkansas • Atlanta, Georgia
Conditions
Duchenne Muscular Dystrophy
Interventions
HT-100
Drug
Lead sponsor
Processa Pharmaceuticals
Industry
Eligibility
6 Years to 20 Years · Male only
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
5
States / cities
Sacramento, California • Baltimore, Maryland • St Louis, Missouri + 2 more
Conditions
Duchenne Muscular Dystrophy Cardiomyopathy, Cardiomyopathy, Dilated
Interventions
Ifetroban, Placebo
Drug
Lead sponsor
Cumberland Pharmaceuticals
Industry
Eligibility
7 Years and older · Male only
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
10
States / cities
Little Rock, Arkansas • Los Angeles, California • Washington D.C., District of Columbia + 7 more
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
AVI-4658 (Eteplirsen)
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 13 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
11
States / cities
Long Beach, California • Gainesville, Florida • Chicago, Illinois + 8 more