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Showing 73–96 of 240 matching trials from the live ClinicalTrials.gov search.
Conditions
Becker Muscular Dystrophy, Sporadic Inclusion Body Myositis
Interventions
rAAV1.CMV.huFollistatin344
Biological
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
1
States / cities
Columbus, Ohio
Conditions
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Limb Girdle Muscular Dystrophy
Interventions
Coenzyme Q10 and Lisinopril
Drug
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
8 Years and older
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
5
States / cities
Washington D.C., District of Columbia • Chicago, Illinois • Charlotte, North Carolina + 2 more
Conditions
DMD-Associated Dilated Cardiomyopathy
Interventions
SRD-001
Genetic
Lead sponsor
Sardocor Corp.
Industry
Eligibility
18 Years and older · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
3
States / cities
Kansas City, Kansas • Cincinnati, Ohio • Columbus, Ohio
Conditions
Duchenne Muscular Dystrophy, Duchenne, DMD, Neuromuscular Diseases, Muscular Dystrophies
Interventions
SAT-3247, Placebo
Drug
Lead sponsor
Satellos Bioscience, Inc.
Industry
Eligibility
7 Years to 9 Years · Male only
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
7
States / cities
Los Angeles, California • Aurora, Colorado • Chicago, Illinois + 4 more
Conditions
Duchenne Muscular Dystrophy
Interventions
Pamrevlumab, Placebo, Corticosteroids
Drug
Lead sponsor
Kyntra Bio
Industry
Eligibility
6 Years to 11 Years · Male only
Enrollment
73 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
27
States / cities
Little Rock, Arkansas • Los Angeles, California • Sacramento, California + 24 more
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
2 Years to 17 Years · Male only
Enrollment
43 participants
Timeline
2017 – 2018
U.S. locations
1
States / cities
Chicago, Illinois
Conditions
Duchenne Muscular Dystrophy
Interventions
RO7239361, Placebo for RO7239361
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
6 Years to 11 Years · Male only
Enrollment
166 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
17
States / cities
Phoenix, Arizona • Palo Alto, California • Sacramento, California + 14 more
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
SGT-003, Placebo
Drug
Lead sponsor
Solid Biosciences Inc.
Industry
Eligibility
7 Years to 11 Years · Male only
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2034
U.S. locations
3
States / cities
Little Rock, Arkansas • Flower Mound, Texas • Norfolk, Virginia
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
Activity Monitor
Device
Lead sponsor
Pfizer
Industry
Eligibility
4 Years to 12 Years · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020
U.S. locations
1
States / cities
Columbus, Ohio
Conditions
Becker Muscular Dystrophy
Interventions
Sodium Nitrate, Sodium Nitrate - double dose, Placebo, Increased exercise intensity
Dietary Supplement · Procedure
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
15 Years to 45 Years · Male only
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Los Angeles, California
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
Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy (DMD)
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
5 Years and older
Enrollment
106 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2028
U.S. locations
3
States / cities
Palo Alto, California • Boston, Massachusetts • New York, New York
Conditions
Duchenne Muscular Dystrophy
Interventions
INS1201
Genetic
Lead sponsor
Insmed Gene Therapy LLC
Industry
Eligibility
2 Years to 4 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
10
States / cities
Little Rock, Arkansas • Davis, California • Los Angeles, California + 7 more
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
Recruiting Not applicable Interventional Accepts healthy volunteers

Urinary Titin Biomarker in DMD

NCT07332013
Conditions
Duchenne Muscular Dystrophy (DMD), Becker's Muscular Dystrophy (BMD)
Interventions
Descending stair walk
Other
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
2 Years to 10 Years · Male only
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2029
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Conditions
Duchenne Muscular Dystrophy
Interventions
Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3, Sevasemten Dose 4, Sevasemten Dose 5, Placebo
Drug
Lead sponsor
Edgewise Therapeutics, Inc.
Industry
Eligibility
4 Years to 9 Years · Male only
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
14
States / cities
Little Rock, Arkansas • Los Angeles, California • Sacramento, California + 11 more
Completed Phase 2 Interventional Results available

Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE)

NCT06304064
Conditions
Duchenne Muscular Dystrophy
Interventions
Allogeneic Cardiosphere-Derived Cells (CAP-1002)
Biological
Lead sponsor
Capricor Inc.
Industry
Eligibility
12 Years and older · Male only
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
2
States / cities
Gainesville, Florida • Cincinnati, Ohio
Conditions
Duchenne Muscular Dystrophy
Interventions
NS-065/NCNP-01
Drug
Lead sponsor
NS Pharma, Inc.
Industry
Eligibility
4 Years to 10 Years · Male only
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
5
States / cities
Sacramento, California • Chicago, Illinois • St Louis, Missouri + 2 more
Conditions
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Interventions
Not listed
Lead sponsor
CureDuchenne
Other
Eligibility
4 Weeks and older
Enrollment
240 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
10
States / cities
Little Rock, Arkansas • Newport Beach, California • Orange, California + 7 more
Conditions
Nonsene Mutation Duchenne Muscular Dystrophy
Interventions
Ataluren
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
6 Months to 2 Years · Male only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Atlanta, Georgia
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
Semaglutide (Rybelsus®), Placebo
Drug · Other
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years and older · Male only
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
Nashville, Tennessee
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Washington University School of Medicine
Other
Eligibility
7 Years to 22 Years · Male only
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
St Louis, Missouri
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