4 completed, 4 recruiting, 3 enrolling by invitation, 1 active not recruiting. 5 Phase 3, 3 Phase 2, 2 Not applicable, 1 Phase 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 |
|---|---|---|---|---|---|---|
| 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 |
| Electromyostimulation (EMS) of Astronauts in Spaceflight (Easymotion-2) Electromyostimulation (EMS), Myoton, Electromyography (EMG) - Muscle Atrophy, Myotonia | Not applicable | Enrolling by invitation | Charite University, Berlin, Germany | 12 | 2025-09-15 | 2026-03-23 |
| 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 |
| Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension Congenital Myotonic Dystrophy, Childhood Myotonic Dystrophy | Recruiting | Virginia Commonwealth University | 200 | 2025-06-06 | 2026-08-11 | |
| A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I VX-670 - Myotonic Dystrophy Type 1 (DM1) | Phase 2 | Enrolling by invitation | Vertex Pharmaceuticals Incorporated | 44 | 2025-04-28 | 2026-10-14 |
| 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 |
| A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) VX-670, Placebo - Myotonic Dystrophy Type 1 (DM1) | Phase 1 / Phase 2 | Active not recruiting | Vertex Pharmaceuticals Incorporated | 47 | 2024-02-20 | 2026-08-20 |
| Assessing Clinical Endpoints and Biomarkers in Myotonic Dystrophy Type-1 and Type 2 (ASCEND-DM) Myotonic Dystrophy Type-1, Myotonic Dystrophy Type-2 | Terminated | National Institute of Neurological Disorders and Stroke (NINDS) | 4 | 2020-02-24 | 2021-11-04 | |
| 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 |
| Effectiveness of Mexiletine for Treating People With Non-Dystrophic Myotonia Mexiletine, Placebo - Myotonia, Non-Dystrophic Myotonia | Phase 2 | Completed | Richard Barohn, MD | 59 | 2008-12-01 | 2013-08-23 |
| Cardiovascular Consequences of NIV Withdrawal in Patients With Myotonic Dystrophy withdrawal of non-invasive ventilation - Myotonia | Not applicable | Unknown | University Hospital, Grenoble | 35 | 2008-06-01 | 2012-08-13 |
| Characteristics of Nondystrophic Myotonias Nondystrophic Myotonias, Myotonia Congenita | Completed | Richard Barohn, MD | 94 | 2006-02-01 | 2013-03-06 |
Myotonic Dystrophy (8) · Myotonic Disorders (7) · Muscular Dystrophies (4) · Neuromuscular Diseases (4) · Genetic Diseases, Inborn (2) · Heredodegenerative Disorders, Nervous System (2) · Muscular Diseases (2) · Muscular Disorders, Atrophic (2) · Musculoskeletal Diseases (2) · Myotonia Congenita (2) · Nervous System Diseases (2) · Neurodegenerative Diseases (2) · Muscle Weakness (1) · Muscular Atrophy (1)
Neurologic Manifestations (14) · Neuromuscular Manifestations (14) · Pathological Conditions, Signs and Symptoms (14) · Signs and Symptoms (14) · Nervous System Diseases (12) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (10) · Genetic Diseases, Inborn (8) · Muscular Diseases (8) · Musculoskeletal Diseases (8) · Heredodegenerative Disorders, Nervous System (7) · Muscular Disorders, Atrophic (7) · Neurodegenerative Diseases (7) · Neuromuscular Diseases (6) · Muscular Dystrophies (5) · Myotonic Disorders (2)
Mexiletine (2) · Electromyography (1) · Lamotrigine (1) · Magnetic Resonance Imaging (1) · Postmortem Imaging (1)
Avidity Biosciences, Inc. (2) · Richard Barohn, MD (2) · Vertex Pharmaceuticals Incorporated (2) · Virginia Commonwealth University (2) · Charite University, Berlin, Germany (1) · Dyne Therapeutics (1) · Grete Andersen, MD (1) · Lupin Ltd. (1) · National Institute of Neurological Disorders and Stroke (NINDS) (1) · University Hospital, Grenoble (1)
United States (10) · United Kingdom (7) · Canada (6) · Germany (6) · France (5) · Italy (5) · Netherlands (5) · Spain (5) · Belgium (4) · Denmark (4) · Australia (3) · Japan (3) · Brazil (1)
Ami K Mankodi, M.D. (1) · Dieter Blottner, PhD (1) · Grete Andersen, MD (1) · Nicholas Johnson, MD (1) · Nicholas Johnson, MD, MSCI, FAAN (1) · Richard Barohn, MD (1)
2023 (1), 2025 (1), R01NS104010 (1)