- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- delandistrogene moxeparvovec, Plasmapheresis
- Genetic · Procedure
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- 4 Years to 8 Years · Male only
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2025
- U.S. locations
- 3
- States / cities
- Gainesville, Florida • St Louis, Missouri • Columbus, Ohio
Search Results
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Showing 25–42
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- Conditions
- Limb Girdle Muscular Dystrophy
- Interventions
- SRP-6004
- Genetic
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years to 50 Years
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2025
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Limb Girdle Muscular Dystrophy
- Interventions
- SRP-9003
- Genetic
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- 4 Years to 50 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2028
- U.S. locations
- 2
- States / cities
- Columbus, Ohio • Memphis, Tennessee
- 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
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Interventions
- eteplirsen
- Drug
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- 4 Years to 6 Years · Male only
- Enrollment
- 33 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2018
- U.S. locations
- 13
- States / cities
- Phoenix, Arizona • Los Angeles, California • Sacramento, California + 9 more
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- AVI-4658 (Eteplirsen), Placebo
- Drug · Other
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- 7 Years to 13 Years · Male only
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2012
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Casimersen, Golodirsen
- Drug
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- 7 Years to 23 Years · Male only
- Enrollment
- 171 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2023
- U.S. locations
- 25
- States / cities
- Phoenix, Arizona • Los Angeles, California • Palo Alto, California + 21 more
- Conditions
- Encephalitis
- Interventions
- AVI-4065 Injection
- Drug
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years to 64 Years · Male only
- Enrollment
- 12 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2006 – 2009
- U.S. locations
- 1
- States / cities
- Tacoma, Washington
- Conditions
- Ebola Hemorrhagic Fever
- Interventions
- AVI-7537, Normal Saline Solution (NSS)
- Drug · Other
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years to 50 Years
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2012 – 2013
- U.S. locations
- 1
- States / cities
- Overland Park, Kansas
- Conditions
- Duchenne Muscular Dystrophy, Burden, Dependency, Disability Physical, Disease Management, Impairment, Rare Diseases
- Interventions
- Not listed
- Lead sponsor
- Jett Foundation, Inc.
- Other
- Eligibility
- 11 Years and older
- Enrollment
- 60 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2019
- U.S. locations
- 1
- States / cities
- Eagan, Minnesota
- Conditions
- Limb-girdle Muscular Dystrophy
- Interventions
- Not listed
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- 4 Years and older
- Enrollment
- 205 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2030
- U.S. locations
- 11
- States / cities
- Phoenix, Arizona • Little Rock, Arkansas • La Jolla, California + 8 more
- Conditions
- Limb-girdle Muscular Dystrophy
- Interventions
- SRP-9003, Glucocorticoid
- Biological · Drug
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- 4 Years and older
- Enrollment
- 17 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2029
- U.S. locations
- 4
- States / cities
- La Jolla, California • Columbus, Ohio • Philadelphia, Pennsylvania + 1 more
- 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
- Limb-Girdle Muscular Dystrophy, Type 2E
- Interventions
- SRP-9003
- Genetic
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- 4 Years to 15 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2025
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- SRP-4045, SRP-4053, Placebo
- Drug
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- 6 Years to 13 Years · Male only
- Enrollment
- 228 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2025
- U.S. locations
- 23
- States / cities
- Phoenix, Arizona • Los Angeles, California • San Diego, California + 19 more
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Golodirsen 50 MG/1 ML Intravenous Solution [VYONDYS 53]
- Drug
- Lead sponsor
- Rare Disease Research, LLC
- Other
- Eligibility
- 7 Years and older · Male only
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2021
- U.S. locations
- 2
- States / cities
- Atlanta, Georgia • Pittsburgh, Pennsylvania
- Conditions
- Dysferlinopathy
- Interventions
- rAAVrh74.MHCK7.DYSF.DV
- Drug
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2019
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- 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