- Conditions
- Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne, Muscular Dystrophy (DMD), DMD, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Duchenne Type, Muscular Dystrophy, Duchenne and Becker Types, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Genetic Disease, Inborn, Genetic Disease, X-Linked, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neuromuscular Diseases (NMD)
- Interventions
- Zeleciment Rostudirsen (DYNE-251), Placebo
- Drug
- Lead sponsor
- Dyne Therapeutics
- Industry
- Eligibility
- 4 Years to 18 Years · Male only
- Enrollment
- 90 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2032
- U.S. locations
- 1
- States / cities
- Hillsborough, North Carolina
Search Results
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Showing 25–42
of 18
matching trials from the live ClinicalTrials.gov search.
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- Conditions
- DM1, Myotonic Dystrophy 1, Myotonic Dystrophy, Myotonic Dystrophy Type 1 (DM1), Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, Myotonic Muscular Dystrophy
- Interventions
- AOC 1001, Placebo
- Drug
- Lead sponsor
- Avidity Biosciences, Inc.
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 39 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 8
- States / cities
- Los Angeles, California • Palo Alto, California • Denver, Colorado + 5 more
- Conditions
- Myotonic Dystrophy, Type 1 (DM1), Myotonic Dystrophy
- Interventions
- ERX-963, Placebo
- Drug
- Lead sponsor
- Expansion Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2020
- U.S. locations
- 4
- States / cities
- Palo Alto, California • Miami, Florida • Iowa City, Iowa + 1 more
- Conditions
- Myotonic Dystrophy
- Interventions
- SomatoKine/IPLEX
- Drug
- Lead sponsor
- University of Rochester
- Other
- Eligibility
- 21 Years to 60 Years
- Enrollment
- 17 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2005 – 2008
- U.S. locations
- 1
- States / cities
- Rochester, New York
- Conditions
- Muscular Dystrophy, Arrhythmia, Sudden Cardiac Death
- Interventions
- Screening
- Other
- Lead sponsor
- Indiana University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 448 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1996 – 2015
- U.S. locations
- 1
- States / cities
- Indianapolis, Indiana
- Conditions
- Myotonic Dystrophy, Facioscapulohumeral Muscular Dystrophy, Muscular Dystrophy, Myotonic Dystrophy Type 1, Myotonic Dystrophy Type 2, Congenital Myotonic Dystrophy, PROMM (Proximal Myotonic Myopathy), Steinert's Disease, Myotonic Muscular Dystrophy
- Interventions
- Not listed
- Lead sponsor
- University of Rochester
- Other
- Eligibility
- Not listed
- Enrollment
- 3,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2000 – 2028
- U.S. locations
- 1
- States / cities
- Rochester, New York
- Conditions
- Congenital Myotonic Dystrophy, Childhood Myotonic Dystrophy, Myotonic Dystrophy
- Interventions
- Not listed
- Lead sponsor
- Virginia Commonwealth University
- Other
- Eligibility
- 3 Years to 17 Years
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2030
- U.S. locations
- 1
- States / cities
- Richmond, Virginia
- Conditions
- DM1, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonia, Myotonic Dystrophy Type 1 (DM1), Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, Steinert, Myotonic Muscular Dystrophy
- Interventions
- AOC 1001 (del-desiran), Placebo
- Drug
- Lead sponsor
- Avidity Biosciences, Inc.
- Industry
- Eligibility
- 16 Years to 65 Years
- Enrollment
- 159 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 18
- States / cities
- Stanford, California • Denver, Colorado • Gainesville, Florida + 15 more
- Conditions
- Myotonic Dystrophy Type 1
- Interventions
- IONIS-DMPKRx, Placebo
- Drug
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 20 Years to 55 Years
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2016
- U.S. locations
- 8
- States / cities
- Stanford, California • Gainesville, Florida • Kansas City, Kansas + 5 more
- Conditions
- Myotonia Congenita, Paramyotonia Congenita, Myotonic Dystrophy 1
- Interventions
- Ranolazine
- Drug
- Lead sponsor
- Ohio State University
- Other
- Eligibility
- 18 Years to 100 Years
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2017
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Myotonic Dystrophy Type 1
- Interventions
- Not listed
- Lead sponsor
- Stanford University
- Other
- Eligibility
- 8 Years to 17 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2013 – 2016
- U.S. locations
- 1
- States / cities
- Stanford, California
- Conditions
- Myotonic Dystrophy, Congenital Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Dystrophy 2, Dystrophia Myotonica, Dystrophia Myotonica 1, Dystrophia Myotonica 2, Myotonia Dystrophica, Myotonic Dystrophy, Congenital, Myotonic Myopathy, Proximal, PROMM (Proximal Myotonic Myopathy), Proximal Myotonic Myopathy, Steinert Disease, Steinert Myotonic Dystrophy, Steinert's Disease, Myotonia Atrophica
- Interventions
- Patient-entered data
- Other
- Lead sponsor
- Myotonic Dystrophy Foundation
- Other
- Eligibility
- Not listed
- Enrollment
- 3,500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2030
- U.S. locations
- 1
- States / cities
- Oakland, California
- Conditions
- Myotonic Dystrophy 1, Excessive Daytime Sleepiness
- Interventions
- Pitolisant Oral Tablet, Placebo oral tablet
- Drug
- Lead sponsor
- Harmony Biosciences Management, Inc.
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2024
- U.S. locations
- 11
- States / cities
- Irvine, California • Aurora, Colorado • Tampa, Florida + 8 more
- 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
- Myotonic Dystrophy, Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy
- Interventions
- Not listed
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 5 Years and older
- Enrollment
- 465 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015 – 2028
- U.S. locations
- 5
- States / cities
- Boston, Massachusetts • Winston-Salem, North Carolina • Pittsburgh, Pennsylvania
- Conditions
- Myotonic Dystrophy Type 1 (DM1)
- Interventions
- VX-670, Placebo
- Drug
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Industry
- Eligibility
- 18 Years to 64 Years
- Enrollment
- 52 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2027
- U.S. locations
- 8
- States / cities
- San Carlos, California • Gainesville, Florida • Fairway, Kansas + 5 more
- Conditions
- Myotonic Dystrophy 1
- Interventions
- PGN-EDODM1 for infusion, Placebo
- Drug · Other
- Lead sponsor
- PepGen Inc
- Industry
- Eligibility
- 18 Years to 60 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2025
- U.S. locations
- 7
- States / cities
- Irvine, California • Palo Alto, California • Atlanta, Georgia + 4 more
- Conditions
- Myotonic Dystrophy Type-1, Myotonic Dystrophy Type-2
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- 11 Years to 70 Years
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2021
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland