54 completed, 34 recruiting, 13 unknown, 10 enrolling by invitation. 30 Not applicable, 20 Phase 2, 17 Phase 3, 13 Phase 1.
Follow Myotonic Dystrophy: new trials by email Literature Review of Myotonic Dystrophy trials Research Report on Myotonic Dystrophy
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Calcium Channel Blocker in Myotonic Dystrophy Type 1 Amlodipine - Myotonic Dystrophy 1 | Phase 1 | Not yet recruiting | University of Rochester | 20 | 2026-12-07 | 2025-12-09 |
| Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension DM1, Myotonic Dystrophy | Recruiting | Virginia Commonwealth University | 1000 | 2026-10-15 | 2026-10-16 | |
| A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping AOC 1044, Placebo - Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy) | Phase 3 | Recruiting | Avidity Biosciences, Inc. | 70 | 2026-09-01 | 2026-10-13 |
| An Ambispective Natural History Study in Myotonic Dystrophy Patients Linking Retrospective Data Captured From the DM-Scope Registry With a Prospective 24-month Follow-up Period Myotonic Dystrophy 1, Myotonic Dystrophy 2 | Not yet recruiting | Lupin Ltd. | 100 | 2026-08-03 | 2026-07-28 | |
| Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO) Zeleciment Rostudirsen (DYNE-251), Placebo - Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne | Phase 3 | Recruiting | Dyne Therapeutics | 90 | 2026-06-01 | 2026-05-27 |
| Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1 zeleciment basivarsen (DYNE-101), Placebo - Myotonic Dystrophy Type 1 (DM1), DM1 | Phase 3 | Recruiting | Dyne Therapeutics | 150 | 2026-05-14 | 2026-10-13 |
| Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers Gait analysis via video capture - Spinal Muscular Atrophy (SMA), Charcot-Marie-Tooth | Not applicable | Recruiting | Institut de Myologie, France | 30 | 2026-03-03 | 2026-05-19 |
| Evaluation of the Role of miR-1 in the Pathogenesis and as a Biomarker in Muscular Dystrophies and Congenital Myopathies dosage of blood biomarker miR1 - Duchenne / Becker Muscular Dystrophy, Dystrophia Myotonica 1 | Not applicable | Recruiting | University Hospital, Clermont-Ferrand | 104 | 2026-02-11 | 2026-02-25 |
| An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE) PGN-EDODM1 - Myotonic Dystrophy 1 | Phase 2 | Recruiting | PepGen Inc | 48 | 2025-12-23 | 2026-03-30 |
| An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2 Who Have Completed MEX-DM-302 Study. Mexiletine granules for prolonged-release oral suspension - Myotonic Dystrophy | Phase 3 | Enrolling by invitation | Lupin Ltd. | 176 | 2025-09-12 | 2026-01-26 |
| The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period. Assistive gait devices combined with physiotherapy - Inclusion Body Myositis, Myotonic Dystrophy 1 | Not applicable | Enrolling by invitation | LMU Klinikum | 30 | 2025-09-01 | 2025-07-18 |
| A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1 SAR446268 - Myotonic Dystrophy | Phase 1 / Phase 2 | Recruiting | Sanofi | 32 | 2025-07-23 | 2026-09-21 |
| Global Open-Label Extension Study of Del-desiran for the Treatment of DM1 Del-desiran (AOC 1001) - Myotonic Dystrophy Type 1, DM1 | Phase 3 | Enrolling by invitation | Avidity Biosciences, Inc. | 199 | 2025-06-26 | 2026-08-19 |
| Fall Frequency and Factors Affecting Dynamic Balance in Patients With Myotonic Dystrophy Type 1 Myotonic Dystrophy Type 1, Falls | Active not recruiting | Antalya Training and Research Hospital | 41 | 2025-06-20 | 2026-03-25 | |
| Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension Congenital Myotonic Dystrophy, Childhood Myotonic Dystrophy | Recruiting | Virginia Commonwealth University | 200 | 2025-06-06 | 2026-08-11 | |
| The Spanish National Registry for Myotonic Dystrophy Type 1 Patient Registry - Myotonic Dystrophy 1, DM1 | Recruiting | Fundació Institut Germans Trias i Pujol | 3000 | 2025-06-02 | 2026-02-04 | |
| Development of Quantitative Muscle Imaging as a Biomarker of Disease Endpoints in Myotonic Dystrophy Myotonic Dystrophy | Recruiting | Wake Forest University Health Sciences | 75 | 2025-05-15 | 2026-01-23 | |
| A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I VX-670 - Myotonic Dystrophy Type 1 (DM1) | Phase 2 | Enrolling by invitation | Vertex Pharmaceuticals Incorporated | 44 | 2025-04-28 | 2026-10-14 |
| Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1) Music and movement education - Myotonic Dystrophy, Congenital, Myotonic Dystrophy, Type 1 (DM1) | Not applicable | Active not recruiting | Hanns Lochmuller | 13 | 2025-03-25 | 2026-07-28 |
| Investigating Exercise in Myotonic Dystrophy Type 2 (DM2) Exercise - Myotonic Dystrophy 2 | Not applicable | Recruiting | Massachusetts General Hospital | 24 | 2025-03-04 | 2026-10-08 |
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Muscular Dystrophies (18) · Neuromuscular Diseases (18) · Muscular Diseases (14) · Myotonic Disorders (11) · Muscular Dystrophy, Duchenne (10) · Muscular Dystrophy, Facioscapulohumeral (10) · Genetic Diseases, Inborn (9) · Myotonia (8) · Muscular Disorders, Atrophic (6) · Musculoskeletal Diseases (6) · Nervous System Diseases (6) · Heredodegenerative Disorders, Nervous System (5) · Huntington Disease (5) · Neurodegenerative Diseases (5)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (123) · Heredodegenerative Disorders, Nervous System (119) · Neurodegenerative Diseases (119) · Muscular Disorders, Atrophic (118) · Musculoskeletal Diseases (118) · Nervous System Diseases (118) · Genetic Diseases, Inborn (115) · Myotonic Disorders (113) · Muscular Diseases (110) · Muscular Dystrophies (106) · Neuromuscular Diseases (106) · Pathological Conditions, Signs and Symptoms (25) · Neurologic Manifestations (16) · Signs and Symptoms (14) · Pathologic Processes (13)
Mexiletine (4) · tideglusib (4) · Exercise (3) · Neuropsychological Tests (2) · Walk Test (2) · Amlodipine (1) · Blood Specimen Collection (1) · Dental Occlusion (1) · Electrocardiography (1) · Electrocardiography, Ambulatory (1) · Health Records, Personal (1) · IGF-I-IGFBP-3 complex (1) · Insulin-Like Growth Factor I (1) · Lamotrigine (1) · Magnetic Resonance Imaging (1)
University of Rochester (8) · Lupin Ltd. (6) · Virginia Commonwealth University (6) · Avidity Biosciences, Inc. (5) · Institut de Myologie, France (5) · LMU Klinikum (5) · AMO Pharma Limited (4) · Assistance Publique - Hôpitaux de Paris (4) · Massachusetts General Hospital (4) · Dyne Therapeutics (3) · PepGen Inc (3) · Centre Hospitalier Universitaire de Liege (2) · Laval University (2) · McMaster University (2) · Oslo University Hospital (2)
United States (42) · France (30) · Canada (27) · United Kingdom (22) · Germany (20) · Italy (19) · Spain (12) · Netherlands (11) · Belgium (10) · Australia (9) · Denmark (7) · New Zealand (6) · Norway (4) · Japan (3) · China (2)
Nicholas Johnson, MD (4) · Johanna Hamel, MD (2) · John W Day, MD, PhD (2) · Karim Wahbi, MD, PhD (2) · Thurman M. Wheeler, MD (2) · Alla Zozulya-Weidenfeller, PhD (1) · Andreas Barth, MD (1) · Anita Simonds, Pr (1) · Araya Puwanant, MD (1) · Araya Puwanant, MD, MS (1) · Arturo Lopez Castel, Ph.D. (1) · Baziel G van Engelen, MD, PhD (1) · Baziel van Engelen, Prof (1) · Benedikt Schoser, MD (1)
PMID 35794169, PMID 10836341, PMID 11972163, PMID 12351998, PMID 16267188, PMID 16849828, PMID 16900450, PMID 18678823, PMID 19708226, PMID 20837825, PMID 22003934, PMID 22453570, PMID 22578232, PMID 24140416, PMID 24803843, PMID 25753091, PMID 27941019, PMID 28168524, PMID 28397002, PMID 28611030, PMID 28662292, PMID 29934199, PMID 30051542, PMID 30185236, PMID 30390395, PMID 31118244, PMID 31429798, PMID 32373059, PMID 32555262, PMID 32942085, PMID 33884298, PMID 35316212, PMID 36790141, PMID 36804094, PMID 38359368, PMID 38454488, PMID 39555632, PMID 39836447, PMID 40197813, PMID 4047090, PMID 40742628, PMID 41379803, PMID 41707138, PMID 41747205, PMID 563010
2023 (3), U54NS048843 (3), 2016 (2), 2025 (2), K23NS125110 (1), R01HL128743 (1), R01NS104010 (1)