71 completed, 43 recruiting, 15 unknown, 11 enrolling by invitation. 40 Not applicable, 21 Phase 2, 20 Phase 3, 13 Phase 1.
Follow Myotonic Disorders: new trials by email Literature Review of Myotonic Disorders trials Research Report on Myotonic Disorders
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 | 2026-11-09 |
| Metabolomic Biomarkers of Myotonic Dystrophy Myotonic Dystrophy Type 1 | Recruiting | Massachusetts General Hospital | 64 | 2026-10-26 | 2026-11-03 | |
| 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 | |
| Personalized Disease Modeling for Rare Genetic Myopathies Congenital Myopathies, Muscular Dystrophy | Enrolling by invitation | Akron Children's Hospital | 3 | 2026-09-22 | 2026-11-03 | |
| 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 | 2026-11-10 |
| 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 | |
| VirtualPark_Pediatric Dual-task cycling, Standard therapy - Dystrophinopathy, Muscular Dystrophies | Not applicable | Recruiting | Istituto di Sistemi e Tecnologie Industriali Intelligenti per il Manifatturiero Avanzato | 22 | 2026-06-30 | 2026-08-11 |
| 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 | 2026-11-13 |
| 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 | 2026-11-03 |
| 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 |
| Prospective, Long Term, Observational Study (Patient Registry) of Paediatric Myotonic Disorders Mexiletine - Myotonic Disorders | Recruiting | Lupin Ltd. | 10 | 2025-09-30 | 2025-09-23 | |
| 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 | 2026-11-03 |
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Myotonic Dystrophy (126) · Myotonia Congenita (29) · Neuromuscular Diseases (27) · Muscular Dystrophies (24) · Muscular Diseases (22) · Muscular Dystrophy, Duchenne (13) · Muscular Dystrophy, Facioscapulohumeral (10) · Myotonia (10) · Genetic Diseases, Inborn (9) · Musculoskeletal Diseases (8) · Motor Activity (6) · Muscular Disorders, Atrophic (6) · Myopathies, Structural, Congenital (6) · Nervous System Diseases (6)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (152) · Nervous System Diseases (151) · Musculoskeletal Diseases (149) · Heredodegenerative Disorders, Nervous System (147) · Neurodegenerative Diseases (147) · Genetic Diseases, Inborn (144) · Muscular Diseases (135) · Muscular Disorders, Atrophic (131) · Neuromuscular Diseases (130) · Muscular Dystrophies (113) · Pathological Conditions, Signs and Symptoms (30) · Neurologic Manifestations (19) · Signs and Symptoms (19) · Nutritional and Metabolic Diseases (18)
Mexiletine (8) · tideglusib (4) · Exercise (3) · Genetic Testing (2) · Neuropsychological Tests (2) · Physical Therapy Modalities (2) · Walk Test (2) · Albuterol (1) · Amlodipine (1) · Base Sequence (1) · Blood Specimen Collection (1) · Dental Occlusion (1) · Dichlorphenamide (1) · Dosage Forms (1) · Electrocardiography (1)
Lupin Ltd. (8) · University of Rochester (8) · Institut de Myologie, France (7) · Assistance Publique - Hôpitaux de Paris (6) · Virginia Commonwealth University (6) · Avidity Biosciences, Inc. (5) · LMU Klinikum (5) · Massachusetts General Hospital (5) · Radboud University Medical Center (5) · AMO Pharma Limited (4) · Copenhagen University Hospital, Rigshospitalet (4) · Dyne Therapeutics (3) · PepGen Inc (3) · Stanford University (3) · University Hospital, Lille (3)
United States (51) · France (38) · Canada (31) · United Kingdom (24) · Germany (20) · Italy (20) · Netherlands (14) · Denmark (12) · Spain (12) · Belgium (10) · Australia (9) · New Zealand (7) · Norway (4) · Japan (3) · China (2)
Nicholas Johnson, MD (4) · John W Day, MD, PhD (3) · Grete Andersen, MD (2) · Johanna Hamel, MD (2) · Karim Wahbi, MD, PhD (2) · Thurman M. Wheeler, MD (2) · Tina Duong, PT, PhD (2) · Alan Beggs, PhD (1) · Alan H. Beggs, Ph.D. (1) · Alla Zozulya-Weidenfeller, PhD (1) · Andreas Barth, MD (1) · Anita Simonds, Pr (1) · Araya Puwanant, MD (1)
PMID 35794169, PMID 10051637, PMID 10192379, PMID 10508519, PMID 10836341, PMID 11558787, PMID 11972163, PMID 12196661, PMID 12351998, PMID 12601110, PMID 12677001, PMID 15056467, PMID 15236405, PMID 16042307, PMID 16267188, PMID 16849828, PMID 16900450, PMID 17160903, PMID 17537630, PMID 18678823, PMID 19232495, PMID 19346529, PMID 19562689, PMID 19708226, PMID 19944167, PMID 19953533, PMID 20434914, PMID 20837825, PMID 21357678, PMID 22003934, PMID 22453570, PMID 22578232, PMID 24140416, PMID 24803843, PMID 25753091, PMID 25880166, PMID 26608622, PMID 26751952, PMID 27941019, PMID 28168524, PMID 28397002, PMID 28611030, PMID 28662292, PMID 29934199, PMID 30051542, PMID 30185236, PMID 30390395, PMID 30535218, PMID 31118244, PMID 31429798
2023 (3), U54NS048843 (3), 2016 (2), 2025 (2), 2019 (1), 2026A (1), K23NS125110 (1), R01HL128743 (1), R01NS104010 (1), R44AR083316 (1)