- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Drisapersen
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 5 Years to 80 Years · Male only
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
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Showing 1–24
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- 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
- 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
- PTC124
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 5 Years and older · Male only
- Enrollment
- 38 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2007
- U.S. locations
- 3
- States / cities
- Cincinnati, Ohio • Philadelphia, Pennsylvania • Salt Lake City, Utah
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Vamorolone
- Drug
- Lead sponsor
- Catalyst Pharmaceuticals, Inc.
- Industry
- Eligibility
- 2 Years and older · Male only
- Enrollment
- 250 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2032
- U.S. locations
- 31
- States / cities
- Phoenix, Arizona • Little Rock, Arkansas • Loma Linda, California + 27 more
- Conditions
- Becker Muscular Dystrophy, Duchenne Muscular Dystrophy
- Interventions
- Gentamicin
- Drug
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000 – 2001
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
- Interventions
- Ataluren, Placebo
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 7 Years to 16 Years · Male only
- Enrollment
- 230 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2015
- U.S. locations
- 21
- States / cities
- Los Angeles, California • Sacramento, California • Aurora, Colorado + 17 more
- 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
- P-188 NF
- Drug
- Lead sponsor
- Phrixus Pharmaceuticals, Inc.
- Industry
- Eligibility
- 12 Years to 25 Years · Male only
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2021
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Dystrophinopathy, Duchenne Muscular Dystrophy, Becker's Muscular Dystrophy
- Interventions
- L-arginine
- Drug
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 7 Years to 11 Years · Male only
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Muscular Dystrophies, Spinal Muscular Atrophy, Duchenne Muscular Dystrophy, Limb Girdle Muscular Dystrophy, FSHD, Cerebral Palsy, Becker Muscular Dystrophy
- Interventions
- Abilitech Assist
- Device
- Lead sponsor
- AbiliTech Medical Inc.
- Industry
- Eligibility
- 10 Years and older
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2023
- U.S. locations
- 1
- States / cities
- Saint Paul, Minnesota
- 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
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
- Interventions
- Ataluren
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 7 Years to 15 Years · Male only
- Enrollment
- 219 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2018
- U.S. locations
- 21
- States / cities
- Los Angeles, California • Sacramento, California • Stanford, California + 18 more
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- WVE-210201, Placebo
- Drug
- Lead sponsor
- Wave Life Sciences USA, Inc.
- Industry
- Eligibility
- 5 Years to 18 Years · Male only
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2019
- U.S. locations
- 1
- States / cities
- Atlanta, Georgia
- Conditions
- Muscular Dystrophy, Duchenne
- Interventions
- MNK-1411, Placebo
- Drug · Other
- Lead sponsor
- Mallinckrodt ARD LLC
- Industry
- Eligibility
- 4 Years to 8 Years · Male only
- Enrollment
- 44 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2020
- U.S. locations
- 5
- States / cities
- Gulf Breeze, Florida • Atlanta, Georgia • Nashville, Tennessee + 2 more
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
- Interventions
- Ataluren, PLACEBO
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 5 Years and older · Male only
- Enrollment
- 360 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2023
- U.S. locations
- 22
- States / cities
- Phoenix, Arizona • Los Angeles, California • Oakland, California + 19 more
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Dystrophinopathy, Dystrophinopathy Symptomatic Female Carrier, Dystrophinopathy Female Carrier
- Interventions
- Not listed
- Lead sponsor
- The Duchenne Registry
- Other
- Eligibility
- Not listed
- Enrollment
- 10,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2047
- U.S. locations
- 1
- States / cities
- Washington D.C., District of Columbia
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Deflazacort
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- 5 Years and older
- U.S. locations
- 42
- States / cities
- Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 36 more
- Conditions
- Becker Muscular Dystrophy
- Interventions
- Sodium nitrate
- Dietary Supplement
- Lead sponsor
- Cedars-Sinai Medical Center
- Other
- Eligibility
- 15 Years to 55 Years · Male only
- Enrollment
- 11 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2013 – 2014
- U.S. locations
- 2
- States / cities
- Los Angeles, California
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Eteplirsen, Golodirsen, Casimersen
- Drug
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- Male only
- Enrollment
- 300 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2033
- U.S. locations
- 20
- States / cities
- Little Rock, Arkansas • Los Angeles, California • Sacramento, California + 17 more
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Deramiocel (CAP-1002)
- Biological
- Lead sponsor
- Capricor Inc.
- Industry
- Eligibility
- 10 Years and older
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2026
- U.S. locations
- 5
- States / cities
- Sacramento, California • Aurora, Colorado • 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
- Prednisolone
- Drug
- Lead sponsor
- Washington University School of Medicine
- Other
- Eligibility
- 1 Month to 30 Months · Male only
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2017
- U.S. locations
- 6
- States / cities
- Sacramento, California • Orlando, Florida • Chicago, Illinois + 3 more
- 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