3 recruiting, 2 completed, 1 enrolling by invitation, 1 unknown. 3 Phase 3, 1 Not applicable, 1 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 3 | Completed 1, Enrolling by invitation 1, Recruiting 1 |
| Not applicable | 1 | Unknown 1 |
| Phase 1 | 1 | Completed 1 |
| Phase 2 | 1 | Completed 1 |
Follow Steinert Disease: new trials by email Literature Review of Steinert Disease trials Research Report on Steinert Disease
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| Ventilatory Response After Non Invasive Ventilation in Type 1 Myotonic Dystrophy Non Invasive Ventilation. - Myotonic Dystrophy 1, Steinert Disease | Not applicable | Unknown | National Institute of Respiratory Diseases, Mexico | 27 | 2016-09-01 | 2018-07-11 |
| Myotonic Dystrophy Family Registry Patient-entered data - Myotonic Dystrophy, Congenital Myotonic Dystrophy | Recruiting | Myotonic Dystrophy Foundation | 3500 | 2013-02-01 | 2024-11-21 |
Myotonic Dystrophy (7) · Myotonic Disorders (4) · Myotonia (3) · Respiratory Insufficiency (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (7) · Genetic Diseases, Inborn (7) · Heredodegenerative Disorders, Nervous System (7) · Muscular Diseases (7) · Muscular Disorders, Atrophic (7) · Muscular Dystrophies (7) · Musculoskeletal Diseases (7) · Nervous System Diseases (7) · Neurodegenerative Diseases (7) · Neuromuscular Diseases (7) · Myotonic Disorders (3) · Neurologic Manifestations (3) · Neuromuscular Manifestations (3) · Pathological Conditions, Signs and Symptoms (3) · Signs and Symptoms (3)
Avidity Biosciences, Inc. (3) · Dyne Therapeutics (1) · Fundació Institut Germans Trias i Pujol (1) · Myotonic Dystrophy Foundation (1) · National Institute of Respiratory Diseases, Mexico (1)
United States (5) · Spain (4) · Denmark (3) · France (3) · Germany (3) · Italy (3) · Japan (3) · Netherlands (3) · United Kingdom (3) · Canada (2) · Australia (1) · Belgium (1) · Mexico (1)
Arturo Lopez Castel, Ph.D. (1) · Gisela Nogales Gadea, Ph.D. (1) · Li Tai, MD (1) · Martha G Torres Fraga, MD (1) · Rogelio Perez Padilla, MD (1) · Tanya Stevenson, EdD, MPH (1) · Virginia Arechavala-Gomeza, Ph.D. (1)
PMID 10836341, PMID 11972163, PMID 12351998, PMID 24803843, PMID 25753091, PMID 4047090, PMID 41707138, PMID 563010
2025 (1)