4 recruiting, 3 active not recruiting. 2 Phase 1, 2 Phase 2, 2 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 2 | Active not recruiting 2 |
| Phase 2 | 2 | Active not recruiting 2 |
| Phase 3 | 2 | Recruiting 2 |
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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 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 | 2026-11-06 |
| Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1 DYNE-101, Placebo - Myotonic Dystrophy Type 1 (DM1) | Phase 1 / Phase 2 | Active not recruiting | Dyne Therapeutics | 127 | 2022-09-05 | 2026-09-23 |
| Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping DYNE-251, Placebo - Duchenne Muscular Dystrophy (DMD) | Phase 1 / Phase 2 | Active not recruiting | Dyne Therapeutics | 86 | 2022-08-12 | 2026-08-11 |
| Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) FSHD | Recruiting | University of Kansas Medical Center | 450 | 2020-12-15 | 2026-05-07 | |
| Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD FSHD-specific functional rating scale, Electrical Impedance Myography - Facioscapulohumeral Muscular Dystrophy | Active not recruiting | University of Kansas Medical Center | 324 | 2018-03-05 | 2026-09-17 |
Myotonic Dystrophy (4) · Muscular Dystrophies (3) · Muscular Dystrophy, Duchenne (2) · Myotonia (2) · Neuromuscular Diseases (2) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (1) · Genetic Diseases, Inborn (1) · Genetic Diseases, X-Linked (1) · Muscular Dystrophy, Facioscapulohumeral (1) · Myotonic Disorders (1)
United States (7) · United Kingdom (5) · Italy (4) · Australia (3) · France (3) · Germany (3) · Netherlands (3) · Belgium (2) · Canada (2) · New Zealand (2) · Spain (2) · Brazil (1) · Denmark (1) · Ireland (1) · Japan (1)
Jeffrey Statland, MD (2) · Rabi Tawil, MD (2) · Nicholas Johnson, MD (1)
2023 (2), 2025 (2), U01NS101944 (1)