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Showing 1–24 of 237 matching trials from the live ClinicalTrials.gov search.
Conditions
Duchenne Muscular Dystrophy
Interventions
Transdiagnostic Behavioral Sleep Intervention
Behavioral
Lead sponsor
University of Pittsburgh
Other
Eligibility
6 Years to 18 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
2 Years to 30 Years · Male only
Enrollment
73 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2015
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Duchenne Muscular Dystrophy
Interventions
rAAVrh74.MCK.GALGT2, PLACEBO (Saline)
Biological · Other
Lead sponsor
Kevin Flanigan
Other
Eligibility
9 Years and older · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
Columbus, Ohio
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

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

NCT02413450
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
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
DYNE-251, Placebo
Drug
Lead sponsor
Dyne Therapeutics
Industry
Eligibility
4 Years to 16 Years · Male only
Enrollment
86 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2031
U.S. locations
11
States / cities
La Jolla, California • Los Angeles, California • Aurora, Colorado + 8 more
Conditions
Duchenne Muscular Dystrophy
Interventions
Ezutromid
Drug
Lead sponsor
Summit Therapeutics
Industry
Eligibility
5 Years and older · Male only
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
9
States / cities
Los Angeles, California • Aurora, Colorado • Orlando, Florida + 6 more
Conditions
Muscular Dystrophy, Duchenne
Interventions
SRP-5051
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
12 Years and older · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
7
States / cities
Sacramento, California • Gulf Breeze, Florida • Atlanta, Georgia + 4 more
Conditions
Muscular Dystrophy, Duchenne
Interventions
Eteplirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 21 Years · Male only
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
9
States / cities
Los Angeles, California • Sacramento, California • Iowa City, Iowa + 6 more
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
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
Muscular Dystrophy, Duchenne, Ambulatory Care
Interventions
Placebo, Idebenone
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
10 Years to 18 Years · Male only
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
9
States / cities
Sacramento, California • Aurora, Colorado • Gainesville, Florida + 6 more
Conditions
Duchenne Muscular Dystrophy
Interventions
Viltolarsen
Drug
Lead sponsor
NS Pharma, Inc.
Industry
Eligibility
Male only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2032
U.S. locations
4
States / cities
Sacramento, California • Chicago, Illinois • Durham, North Carolina + 1 more
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
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
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
Duchenne Muscular Dystrophy
Interventions
PF-06252616, Placebo
Biological · Drug
Lead sponsor
Pfizer
Industry
Eligibility
6 Years to 15 Years · Male only
Enrollment
121 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
30
States / cities
Los Angeles, California • Sacramento, California • Aurora, Colorado + 14 more
Conditions
Duchene Muscular Dystrophy
Interventions
Not listed
Lead sponsor
ITF Therapeutics LLC
Industry
Eligibility
6 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
9
States / cities
Little Rock, Arkansas • Washington D.C., District of Columbia • Chicago, Illinois + 6 more
Conditions
Muscular Dystrophies
Interventions
GSK2402968 3mg/kg/week, GSK2402968 6 mg/kg/week, Placebo to match GSK2402968 3 mg/kg/week, Placebo to match GSK2402968 6 mg/kg/week
Drug
Lead sponsor
GlaxoSmithKline
Industry
Eligibility
5 Years and older · Male only
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
14
States / cities
Sacramento, California • Stanford, California • Gulf Breeze, Florida + 11 more
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
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
Eplerenone, Spironolactone
Drug
Lead sponsor
Ohio State University
Other
Eligibility
7 Years and older · Male only
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
6
States / cities
Los Angeles, California • Aurora, Colorado • Kansas City, Kansas + 3 more
Completed Not applicable Interventional Accepts healthy volunteers

Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for DMD

NCT02834650
Conditions
Muscular Dystrophy, Duchenne
Interventions
Cardiac MRI with contrast, Cardiac MRI without contrast, Blood Test, Heart Rate, Pulmonary Function Test, Genetic Testing, Repeat MRI scan
Other
Lead sponsor
Stanford University
Other
Eligibility
7 Years to 21 Years · Male only
Enrollment
89 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2022
U.S. locations
3
States / cities
Los Angeles, California • Orange, California • Stanford, California
Conditions
Becker Muscular Dystrophy
Interventions
Vamorolone, Placebo
Drug
Lead sponsor
ReveraGen BioPharma, Inc.
Industry
Eligibility
18 Years to 64 Years · Male only
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Conditions
Duchenne Muscular Dystrophy
Interventions
PF-06939926
Genetic
Lead sponsor
Pfizer
Industry
Eligibility
4 Years and older · Male only
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
22
States / cities
Los Angeles, California • Durham, North Carolina • Salt Lake City, Utah