2 completed, 2 recruiting, 1 enrolling by invitation. 5 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 5 | Completed 2, Recruiting 2, Enrolling by invitation 1 |
Follow Myotonia: new trials by email Literature Review of Myotonia trials Research Report on Myotonia
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 | 2026-11-06 |
| An Open-Label, Randomized, Cross-Over Study to Investigate the Efficacy and Safety of Mexiletine PR Compared to Mexiletine IR Granular powder in unit dose foil-lined sachet Mexiletine (PR), Oral Capsule Mexiletine (IR) - Myotonia | Phase 3 | Recruiting | Lupin Ltd. | 24 | 2025-09-03 | 2025-12-22 |
| 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 |
| 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 |
| Lamotrigine as Treatment of Myotonia Lamotrigine, Placebo - Dystrophia Myotonica Type 1, Myotonia Congenita | Phase 3 | Completed | Grete Andersen, MD | 27 | 2013-11-01 | 2016-04-25 |
Myotonic Disorders (4) · Myotonic Dystrophy (4) · Myotonia Congenita (1) · Paralysis, Hyperkalemic Periodic (1) · Potassium aggravated myotonia (1)
Nervous System Diseases (5) · Neurologic Manifestations (5) · Neuromuscular Manifestations (5) · Pathological Conditions, Signs and Symptoms (5) · Signs and Symptoms (5) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Genetic Diseases, Inborn (4) · Heredodegenerative Disorders, Nervous System (4) · Muscular Diseases (4) · Muscular Disorders, Atrophic (4) · Muscular Dystrophies (4) · Musculoskeletal Diseases (4) · Neurodegenerative Diseases (4) · Neuromuscular Diseases (4) · Metabolic Diseases (1)
Lamotrigine (1) · Mexiletine (1)
Avidity Biosciences, Inc. (2) · Dyne Therapeutics (1) · Grete Andersen, MD (1) · Lupin Ltd. (1)
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