5 recruiting, 4 completed, 1 enrolling by invitation. 3 Phase 3, 2 Phase 2, 1 Not applicable, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 3 | Completed 1, Enrolling by invitation 1, Recruiting 1 |
| Phase 2 | 2 | Completed 2 |
| Not applicable | 1 | Completed 1 |
| Phase 1 | 1 | Completed 1 |
Follow DM1: new trials by email Literature Review of DM1 trials Research Report on DM1
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 |
|---|---|---|---|---|---|---|
| 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 | |
| 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 |
| 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 | |
| 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 |
| 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 |
| Remote Assessments and Genetic Determinants of Myotonic Dystrophy Myotonic Dystrophy Type 1 (DM1), DM1 | Recruiting | University of Rochester | 1000 | 2022-05-10 | 2026-04-01 | |
| Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients AOC 1001, Placebo - DM1, Myotonic Dystrophy 1 | Phase 1 / Phase 2 | Completed | Avidity Biosciences, Inc. | 39 | 2021-10-28 | 2026-09-28 |
| Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Myotonic Dystrophy 1, DM1 | Recruiting | Virginia Commonwealth University | 700 | 2019-01-01 | 2026-06-10 | |
| Induction of Pluripotent Stem Cells From Human Fibroblasts of DM1 Patients Clinical examination, skin biopsy, blood and urine sampling - DM1 | Not applicable | Completed | Institut de Myologie, France | 12 | 2013-11-01 | 2014-09-26 |
Myotonic Dystrophy (9) · Myotonic Disorders (5) · Myotonia (4) · Muscular Dystrophies (2) · Neuromuscular Diseases (2) · Genetic Diseases, Inborn (1) · Heart Diseases (1) · Heredodegenerative Disorders, Nervous System (1) · Muscular Diseases (1) · Muscular Disorders, Atrophic (1) · Musculoskeletal Diseases (1) · Nervous System Diseases (1) · Neurodegenerative Diseases (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (9) · Genetic Diseases, Inborn (8) · Heredodegenerative Disorders, Nervous System (8) · Muscular Diseases (8) · Muscular Disorders, Atrophic (8) · Musculoskeletal Diseases (8) · Nervous System Diseases (8) · Neurodegenerative Diseases (8) · Muscular Dystrophies (7) · Neuromuscular Diseases (7) · Myotonic Disorders (4) · Neurologic Manifestations (4) · Neuromuscular Manifestations (4) · Pathological Conditions, Signs and Symptoms (4) · Signs and Symptoms (4)
Blood Specimen Collection (1) · Restraint, Physical (1) · Sodium Chloride (1)
Avidity Biosciences, Inc. (4) · Virginia Commonwealth University (2) · Dyne Therapeutics (1) · Fundació Institut Germans Trias i Pujol (1) · Institut de Myologie, France (1) · University of Rochester (1)
United States (8) · France (4) · Germany (4) · Italy (4) · Netherlands (4) · Spain (4) · United Kingdom (4) · Canada (3) · Denmark (3) · Japan (3) · Australia (1) · Belgium (1) · New Zealand (1)
Nicholas Johnson, MD (2) · Arturo Lopez Castel, Ph.D. (1) · Charles Thornton, MD (1) · Gisela Nogales Gadea, Ph.D. (1) · Johanna Hamel, MD (1) · Laurent Servais, MD PhD (1) · Li Tai, MD (1) · Virginia Arechavala-Gomeza, Ph.D. (1)
PMID 39836447, PMID 41379803, PMID 41707138, PMID 41747205
2025 (1)