- Conditions
- Healthy, Muscular Dystrophy, Retinal Degeneration, Skin Diseases, Virus Diseases
- Interventions
- Not listed
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- Not listed
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 1978 – 2000
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
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Showing 25–48
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- Conditions
- Muscular Dystrophies, Cardiac Fibrosis, Genetic Diseases, Inborn, Musculoskeletal Diseases
- Interventions
- Not listed
- Lead sponsor
- Children's National Research Institute
- Other
- Eligibility
- 6 Years and older
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2024
- U.S. locations
- 1
- States / cities
- Washington D.C., District of Columbia
- Conditions
- All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis, Asthma, Atopic Dermatitis Eczema, Beta Thalassemia, Bipolar Disorder, Breast Cancer, Celiac Disease, Cervical Cancer, Chronic Inflammatory Demyelinating Polyneuropathy, Chronic Kidney Diseases, Chronic Obstructive Pulmonary Disease, Colon Cancer, Colorectal Cancer, Crohn's Disease, Cystic Fibrosis, Depression, Diabetes Mellitus, Duchenne Muscular Dystrophy, Endometriosis, Epilepsy, Facioscapulohumeral Muscular Dystrophy, G6PD Deficiency, General Anxiety Disorder, Hepatitis B, Hereditary Hemorrhagic Telangiectasia, HIV/AIDS, Human Papilloma Virus, Huntington's Disease, Idiopathic Thrombocytopenic Purpura, Insomnia, Kidney Cancer, Leukemia, Lung Cancer, Lupus Nephritis, Lymphoma, Melanoma, Multiple Myeloma, Multiple Sclerosis, Myositis, Myotonic Dystrophy, Ovarian Cancer, Pancreatic Cancer, Parkinson's Disease, Polycystic Kidney Diseases, Prostate Cancer, Psoriasis, Psoriatic Arthritis, Rosacea, Scleroderma, Sickle Cell Anemia, Sickle Cell Trait, Sjogren's Syndrome, Skin Cancer, Spinal Muscular Atrophy, Systemic Lupus Erythematosus, Thrombotic Thrombocytopenic Purpura, Trisomy 21, Ulcerative Colitis
- Interventions
- Not listed
- Lead sponsor
- Sanguine Biosciences
- Industry
- Eligibility
- 18 Years to 100 Years
- Enrollment
- 17,667 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2013 – 2024
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- 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
- Becker Muscular Dystrophy
- Interventions
- Sevasemten
- Drug
- Lead sponsor
- Edgewise Therapeutics, Inc.
- Industry
- Eligibility
- Male only
- Enrollment
- 260 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2031
- U.S. locations
- 24
- States / cities
- Little Rock, Arkansas • La Jolla, California • Los Angeles, California + 21 more
- Conditions
- Becker Muscular Dystrophy
- Interventions
- Not listed
- Lead sponsor
- Cooperative International Neuromuscular Research Group
- Network
- Eligibility
- 4 Years and older · Male only
- Enrollment
- 85 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2018
- U.S. locations
- 13
- States / cities
- Sacramento, California • Washington D.C., District of Columbia • Gainesville, Florida + 10 more
- 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
- Facioscapulohumeral Muscular Dystrophy
- Interventions
- EPI-321
- Biological
- Lead sponsor
- Epicrispr Biotechnologies, Inc.
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2032
- U.S. locations
- 5
- States / cities
- Los Angeles, California • Atlanta, Georgia • Baltimore, Maryland + 2 more
- Conditions
- Incomplete Spinal Cord Injury, Muscular Dystrophy, Spina Bifida, Cerebral Palsy
- Interventions
- EA-KAFO
- Device
- Lead sponsor
- National Institutes of Health Clinical Center (CC)
- NIH
- Eligibility
- 5 Years to 100 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2025
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Freeman-Sheldon Syndrome, Arthrogryposis Distal Type 2A, Whistling Face Syndrome, Craniocarpotarsal Dysplasia, Craniocarpotarsal Dystrophy, Freeman-Sheldon Syndrome Variant, Sheldon-Hall Syndrome, Arthrogryposis Distal Type 2B, Gordon Syndrome, Arthrogryposis Distal Type 3, Arthrogryposis Distal Type 1, Arthrogryposis, Distal, Type 1A, Arthrogryposis Distal Type 1B, Arthrogryposis, Distal, Craniofacial Abnormalities, Arthrogryposis
- Interventions
- PTSD Checklist-Civilian (PCL-C), Modified Flanagan Quality of Life Scale, Center for Epidemiologic Studies Depression Scale (CES-D), Functional Enquiry (or Review of Systems) Form, Study of Therapeutic Outcomes and Practices in Freeman-Sheldon Syndrome (STOP-FSS) Questionnaire, FSRG Semi-Structured Quality of Life Interview (FSRG SSQLI), Medical Records Review
- Other
- Lead sponsor
- Freeman-Sheldon Research Group, Inc.
- Other
- Eligibility
- Not listed
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2022
- U.S. locations
- 1
- States / cities
- Buckhannon, West Virginia
- Conditions
- Healthy Volunteer, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn
- Interventions
- EDG-5506 Tablet, Radiolabeled EDG-5506 Suspension, Radiolabeled EDG-5506 Intravenous
- Drug
- Lead sponsor
- Edgewise Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years to 55 Years · Male only
- Enrollment
- 15 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023
- U.S. locations
- 1
- States / cities
- Madison, Wisconsin
- Conditions
- Duchenne Muscular Dystrophy, Duchenne Disease, Muscular Dystrophy, Muscular Dystrophy in Children, Vasodilation, Exercise, DMD
- Interventions
- Tadalafil, Placebo, Exercise Training
- Drug · Other
- Lead sponsor
- University of Florida
- Other
- Eligibility
- 6 Years and older · Male only
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 1
- States / cities
- Gainesville, Florida
- Conditions
- Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Muscular Dystrophies, Muscular Dystrophy, Duchenne
- Interventions
- delpacibart zotadirsen
- Drug
- Lead sponsor
- Avidity Biosciences, Inc.
- Industry
- Eligibility
- 2 Years and older · Male only
- U.S. locations
- 21
- States / cities
- Birmingham, Alabama • Little Rock, Arkansas • Irvine, California + 17 more
- Conditions
- Myotonic Dystrophy Type 1
- Interventions
- Not listed
- Lead sponsor
- University of Rochester
- Other
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 120 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2017
- U.S. locations
- 6
- States / cities
- Stanford, California • Gainesville, Florida • Kansas City, Kansas + 3 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
- 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
- DM1, Muscular Dystrophies, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Disorders, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, Neurodegenerative Diseases
- Interventions
- Placebo, AOC 1001
- Drug
- Lead sponsor
- Avidity Biosciences, Inc.
- Industry
- Eligibility
- 18 Years to 66 Years
- Enrollment
- 37 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2025
- U.S. locations
- 8
- States / cities
- Los Angeles, California • Palo Alto, California • Denver, Colorado + 5 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
- Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, PLS, Motor Neuron Disease, Lou Gehrigs Disease, Familial Disease, Amyotrophic Lateral Sclerosis, Sporadic, Muscular Dystrophy, Miyoshi Myopathy, Amyotrophic Lateral Sclerosis With Dementia
- Interventions
- Not listed
- Lead sponsor
- University of Massachusetts, Worcester
- Other
- Eligibility
- Not listed
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2009 – 2024
- U.S. locations
- 1
- States / cities
- Worcester, Massachusetts
- Conditions
- Oculopharyngeal Muscular Dystrophy
- Interventions
- BB-301: Dose escalation phase 1b cohort 1, BB-301: Dose escalation phase 1b cohort 2, BB-301: Dose escalation phase 1b cohort 3, BB-301: Dose expansion phase 2a
- Genetic
- Lead sponsor
- Benitec Biopharma, Inc.
- Industry
- Eligibility
- Up to 65 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2040
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- idebenone 150 mg film-coated tablets
- Drug
- Lead sponsor
- Santhera Pharmaceuticals
- Industry
- Eligibility
- 11 Years and older · Male only
- Enrollment
- 161 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2020
- U.S. locations
- 13
- States / cities
- Birmingham, Alabama • Tucson, Arizona • Los Angeles, California + 10 more
- Conditions
- Muscular Dystrophies, Limb-Girdle
- Interventions
- SRP-9005, Corticosteroid
- Biological · Drug
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- 4 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2032
- U.S. locations
- 1
- States / cities
- Norfolk, Virginia
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- AAV8 DetectCDx
- Diagnostic Test
- Lead sponsor
- REGENXBIO Inc.
- Industry
- Eligibility
- 0 Years to 25 Years · Male only
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2026
- U.S. locations
- 1
- States / cities
- Atlanta, Georgia
- Conditions
- Glycogen Storage Disease, GSD Type 0A, GSD Type 0B, GSD VII, Tarui Disease, GSD X, GSD XII, GSD XIII, GSD XV, PGBM2, PRKAG2, Danon Disease, Polyglucosan Body Myopathy Type 1, Polyglucosan Body Myopathy Type 2, RBCK1 Deficiency
- Interventions
- No intervention
- Other
- Lead sponsor
- Duke University
- Other
- Eligibility
- 0 Years to 90 Years
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2034
- U.S. locations
- 1
- States / cities
- Durham, North Carolina