- 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
Search Results
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Showing 25–47
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- Conditions
- Myotonic Dystrophy Type 1, DM1, Myotonic Dystrophy, Myotonia, Myotonic Dystrophy 1, Myotonic Disorders, Steinert Myotonic Dystrophy, Steinert Disease
- Interventions
- Del-desiran (AOC 1001)
- Drug
- Lead sponsor
- Avidity Biosciences, Inc.
- Industry
- Eligibility
- 16 Years and older
- Enrollment
- 199 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2030
- U.S. locations
- 19
- States / cities
- Los Angeles, California • Stanford, California • Denver, Colorado + 16 more
- 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
- Myotonic Dystrophy Type 1 (DM1), DM1
- Interventions
- Not listed
- Lead sponsor
- University of Rochester
- Other
- Eligibility
- 18 Years to 88 Years
- Enrollment
- 1,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2030
- U.S. locations
- 1
- States / cities
- Rochester, New York
- 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
- 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
- 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
- Myopathy, Muscular Dystrophies, Myositis, Myofibrillar Myopathy, Congenital Myopathy, Distal Myopathy, Myopathies
- Interventions
- Electrical Impedance Myography
- Device
- Lead sponsor
- Beth Israel Deaconess Medical Center
- Other
- Eligibility
- 18 Years to 89 Years
- Enrollment
- 150 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025 – 2027
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Myotonic Dystrophy Type 2, Myotonic Dystrophy Type 1
- Interventions
- Non-interventional study
- Other
- Lead sponsor
- Wake Forest University Health Sciences
- Other
- Eligibility
- 30 Years to 65 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023 – 2027
- U.S. locations
- 1
- States / cities
- Winston-Salem, North Carolina
- Conditions
- Myotonic Dystrophy 1
- Interventions
- Not listed
- Lead sponsor
- Stanford University
- Other
- Eligibility
- 12 Years to 90 Years
- Enrollment
- 125 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2024
- U.S. locations
- 1
- States / cities
- Palo Alto, California
- Conditions
- Central Core Disease, Centronuclear Myopathy, Congenital Fiber Type Disproportion, Multiminicore Disease, Myotubular Myopathy, Nemaline Myopathy, Rigid Spine Muscular Dystrophy, Undefined Congenital Myopathy
- Interventions
- Not listed
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 4,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2050
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- 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
- 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
- Myotonic Dystrophy Type 1, Congenital DM1, Juvenile DM1, Childhood DM1
- Interventions
- Not listed
- Lead sponsor
- University of Rochester
- Other
- Eligibility
- 0 Years to 17 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2030
- U.S. locations
- 1
- States / cities
- Rochester, New York
- Conditions
- LGMD1B, LGMD1C, LGMD1D, LGMD1E, LGMD1F, LGMD1G, LGMD1H, LGMD2A, LGMD2B, LGMD2C, LGMD2D, LGMD2E, LGMD2F, LGMD2G, LGMD2I, LGMD2J, LGMD2K, LGMD2L, LGMD2M, LGMD2N, LGMD2O, LGMD2P, LGMD2Q, LGMD2S, LGMD2T, LGMD2U, LGMD2W, LGMD2X, LGMD2Y
- Interventions
- Not listed
- Lead sponsor
- Virginia Commonwealth University
- Other
- Eligibility
- 6 Years to 50 Years
- Enrollment
- 1,000 participants
- Timeline
- 2023 – 2029
- U.S. locations
- 1
- States / cities
- Richmond, Virginia
- 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
- 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
- Conditions
- Myotonic Dystrophy
- Interventions
- Not listed
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 5 Years and older
- Enrollment
- 215 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2027
- U.S. locations
- 3
- States / cities
- Boston, Massachusetts • Dallas, Texas
- Conditions
- Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency, Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Fatty Liver and Infantile-onset Cataract Caused by TRAPPC11 Mutations), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan and Epilepsy), Alpha-Dystroglycanopathy (Dystroglycanopathy, Congenital With or Without Mental Retardation (Formerly MDC1C)), Alpha-Dystroglycanopathy (Fukuyama CMD), Alpha-Dystroglycanopathy (LGMDR09 FKRP Related (Formerly LGMD2I)), Alpha-Dystroglycanopathy (LGMDR11 POMT1 Related (Formerly LGMD2K)), Alpha-Dystroglycanopathy (LGMDR13 FKTN Related (Formerly LGMD2M)), Alpha-Dystroglycanopathy (LGMDR14 POMT2 Related (Formerly LGMD2N)), Alpha-Dystroglycanopathy (LGMDR15 POMGnT1 Related (Formerly LGMD2O)), Alpha-Dystroglycanopathy (LGMDR19 GMPPB Related (Formerly LGMD2T)), Alpha-Dystroglycanopathy (LGMDR20 ISPD Related (Formerly LGMD2U)), Alpha-Dystroglycanopathy (LGMDR24 POMGnT2 Related), Alpha-Dystroglycanopathy (Muscle Eye Brain Disease (MEB)), Alpha-Dystroglycanopathy (Walker Warburg Syndrome (WWS)), Choline Kinase B Receptor - CHKB, Collagen VI Related Disorders, Collagen XII Related Disorders, Congenital Muscular Dystrophy Not Otherwise Specified (Including Merosin Positive), Congenital Muscular Dystrophy With Cataracts and Intellectual Disability (MDCCAID), Congenital Muscular Dystrophy With Joint Hyperlaxity, Congenital Muscular Dystrophy With Rigid Spine Related to ACTA1, Emery-Dreifuss Muscular Dystrophy, GOLGA2-related Congenital Muscle Dystrophy With Brain Involvement, LMNA Related Disorders, Merosin Deficient CMD (Full or Partial), Nesprin Related MD (SYNE1), SELENON Related Disorders (Previously Known as SEPN1), SELENON Related Myopathy (Aka SEPN1), Telethonin CMD, Congenital Myasthenic Syndrome, Limb-Girdle Muscular Dystrophy, LGMDD01 - DNAJB6 (Formerly LGMD1D), LGMDD05 - Collagen VI Related Bethlem Myopathy (Dominant), LGMDR07 - Telethonin (TCAP) Related (Formerly LGMD2G), LGMDR08 - TRIM Related (Formerly LGMD2H), LGMDR09 - FKRP Related (Formerly LGMD2I), LGMDR10 - Titin (TTN) Related (Formerly LGMD2J), LGMDR11 - POMT1 Related (Formerly LGMD2K), LGMDR13 - Fukutin (FKTN) Related (Formerly LGMD2M), LGMDR14 - POMT2 Related (Formerly LGMD2N), LGMDR15 - POMGnT1 Related (Formerly LGMD2O), LGMDR16 - DAG1 Related Dystroglycanopathy (Formerly LGMD2P), LGMDR17 - Plectin (PLEC) Related (Formerly LGMD2Q), LGMDR18 - TRAPPC11 Related (Formerly LGMD2S), LGMDR19 - GMPPB Related (Formerly LGMD2T), LGMDR20 - ISPD Related (Formerly LGMD2U), LGMDR22 - Collagen VI Related Bethlem Myopathy (Recessive), LGMDR23 - LAMA2 Related, LGMDR24 - POMGnT2 Related
- Interventions
- Not listed
- Lead sponsor
- Cure CMD
- Other
- Eligibility
- Not listed
- Enrollment
- 4,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2029
- U.S. locations
- 1
- States / cities
- Lakewood, California
- 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 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 (DM1)
- Interventions
- VX-670
- Drug
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 44 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2029
- U.S. locations
- 2
- States / cities
- St Louis, Missouri • Richmond, Virginia
- Conditions
- DM1, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Dystrophy Type 1, Myotonic Dystrophy Type-1, Myotonic Dystrophy, Type 1 (DM1), Myotonic Muscular Dystrophy
- Interventions
- Not listed
- Lead sponsor
- Virginia Commonwealth University
- Other
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 1,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2032
- U.S. locations
- 1
- States / cities
- Richmond, Virginia