21 completed, 10 recruiting, 6 unknown, 4 enrolling by invitation. 14 Not applicable, 8 Phase 2, 6 Phase 1, 4 Phase 3.
Follow Myotonic Dystrophy 1: new trials by email Literature Review of Myotonic Dystrophy 1 trials Research Report on Myotonic Dystrophy 1
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 | |
| 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 | |
| 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 |
| 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 |
| 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 |
| 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 | |
| A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 PGN-EDODM1, Placebo - Myotonic Dystrophy 1 | Phase 2 | Recruiting | PepGen Inc | 24 | 2024-12-10 | 2026-07-29 |
| Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease) Treatment taken - Steinert's Disease, Myotonic Dystrophy 1 | Phase 3 | Recruiting | Assistance Publique - Hôpitaux de Paris | 142 | 2024-11-29 | 2025-11-24 |
| Study of ATX-01 in Participants With DM1 ATX-01, Placebo - Myotonic Dystrophy 1 | Phase 1 / Phase 2 | Recruiting | ARTHEx Biotech S.L. | 56 | 2024-10-15 | 2026-02-10 |
| Global Study of Del-desiran for the Treatment of DM1 AOC 1001 (del-desiran), Placebo - DM1, Myotonic Dystrophy | Phase 3 | Completed | Avidity Biosciences, Inc. | 159 | 2024-05-30 | 2026-09-01 |
| Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology Syde, Dynamometric measurements of muscle strength, The 6-minute walk test (6MWT) - Neuromuscular Diseases, Obesity (Disorder) | Not applicable | Recruiting | Centre Hospitalier Universitaire de Liege | 300 | 2024-03-29 | 2025-08-22 |
| Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy SRP-1003 IV Infusion, Placebo IV Infusion, SRP-1003 SC Injection - Myotonic Dystrophy 1 | Phase 1 / Phase 2 | Enrolling by invitation | Sarepta Therapeutics, Inc. | 78 | 2024-03-04 | 2026-10-05 |
| Home-based Training and Supplementation in DM1 Patients Mult-ingredient supplement, Placebo, Concurrent exercise training - Myotonic Dystrophy 1 | Phase 3 | Unknown | McMaster University | 60 | 2024-01-01 | 2023-10-19 |
| Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1 PGN-EDODM1 for infusion, Placebo - Myotonic Dystrophy 1 | Phase 1 | Completed | PepGen Inc | 24 | 2023-12-12 | 2026-02-13 |
| Evaluation of Cognitive Functions in 20 Patients With Type 1 Myotonic Dystrophy With Virtual Reality Approach Type 1 myotonic dystrophy - Myotonic Dystrophy 1 | Not applicable | Withdrawn | Centre Hospitalier Universitaire de Nice | 2023-11-01 | 2024-02-21 | |
| Profile of Dysphagia in Myotonic Dystrophy Type 1 (DM1) Myotonic Dystrophy 1, Dysphagia, Oropharyngeal | Completed | University College, London | 177 | 2023-07-03 | 2025-08-19 | |
| Myotonic Dystrophy Type 1 Congenital and Juvenile Form: From Diagnosis to Rehabilitation [MDCJ-NeuBeRe] neurocognitive evaluations - Myotonic Dystrophy 1 | Unknown | IRCCS Eugenio Medea | 30 | 2022-09-15 | 2024-04-22 | |
| Observational Study of Digital Biomarkers of Myotonia and Gait in Adults and Children With Myotonic Dystrophy Myotonic Dystrophy 1 | Unknown | Stanford University | 125 | 2022-09-10 | 2023-10-18 | |
| Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients Placebo, AOC 1001 - DM1, Muscular Dystrophies | Phase 2 | Completed | Avidity Biosciences, Inc. | 37 | 2022-08-04 | 2026-09-11 |
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Myotonic Dystrophy (45) · Myotonic Disorders (7) · Neuromuscular Diseases (7) · Muscular Diseases (4) · Muscular Dystrophies (4) · Genetic Diseases, Inborn (3) · Heredodegenerative Disorders, Nervous System (3) · Muscular Disorders, Atrophic (3) · Muscular Dystrophy, Facioscapulohumeral (3) · Musculoskeletal Diseases (3) · Myotonia (3) · Nervous System Diseases (3) · Charcot-Marie-Tooth Disease (2) · Huntington Disease (2) · Lambert-Eaton Myasthenic Syndrome (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (45) · Neurodegenerative Diseases (43) · Genetic Diseases, Inborn (42) · Heredodegenerative Disorders, Nervous System (42) · Muscular Disorders, Atrophic (42) · Musculoskeletal Diseases (42) · Nervous System Diseases (42) · Muscular Diseases (41) · Muscular Dystrophies (41) · Myotonic Disorders (38) · Neuromuscular Diseases (38) · Pathological Conditions, Signs and Symptoms (7) · Neurologic Manifestations (6) · Signs and Symptoms (5) · Congenital Abnormalities (4)
Neuropsychological Tests (2) · Amlodipine (1) · Electrocardiography (1) · Electrocardiography, Ambulatory (1) · Exercise (1) · Health Records, Personal (1) · Magnetic Resonance Imaging (1) · Noninvasive Ventilation (1) · Physical Therapy Modalities (1) · Ranolazine (1) · Respiratory Function Tests (1) · Sodium Chloride (1) · Videotape Recording (1) · Walk Test (1) · pitolisant (1)
Avidity Biosciences, Inc. (4) · PepGen Inc (3) · Assistance Publique - Hôpitaux de Paris (2) · Centre Hospitalier Universitaire de Liege (2) · LMU Klinikum (2) · McMaster University (2) · Virginia Commonwealth University (2) · AMO Pharma Limited (1) · ARTHEx Biotech S.L. (1) · Centre Hospitalier Universitaire de Nice (1) · Cynthia Gagnon (1) · Fundació Institut Germans Trias i Pujol (1) · Harmony Biosciences Management, Inc. (1) · Huashan Hospital (1) · IRCCS Eugenio Medea (1)
Canada (14) · United States (12) · France (9) · United Kingdom (9) · Italy (8) · Germany (6) · Spain (6) · Belgium (4) · Netherlands (4) · New Zealand (3) · Denmark (2) · Japan (2) · Norway (2) · Australia (1) · China (1)
Karim Wahbi, MD, PhD (2) · Nicholas Johnson, MD (2) · Alla Zozulya-Weidenfeller, PhD (1) · Arturo Lopez Castel, Ph.D. (1) · Bernd Weber, Prof. Dr. (1) · Charles Thornton, MD (1) · Chongbo Zhao, PhD (1) · Cornelia Kornblum, Prof. Dr. (1) · Cynthia Gagnon, Ph.D. (1) · Denis Duboc, MD, PhD (1) · Denis Furling, Md, PhD (1) · Elise Duchesne, Ph.D (1) · Elise Duchesne, Ph.D. (1) · Gisela Nogales Gadea, Ph.D. (1)
PMID 10836341, PMID 11972163, PMID 12351998, PMID 24803843, PMID 25753091, PMID 30390395, PMID 31429798, PMID 32942085, PMID 35316212, PMID 35794169, PMID 36790141, PMID 38454488, PMID 39836447, PMID 40197813, PMID 4047090, PMID 41379803, PMID 41707138, PMID 41747205, PMID 563010
2016 (1), 2023 (1)