Myotonia clinical trials
The registry lists 17 studies for Myotonia across more than 10 countries. 7 are recruiting or about to start. 5 of them are mainly about Myotonia (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-09.
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Status
Who runs them
Virginia Commonwealth University (2) · Vertex Pharmaceuticals Incorporated (2) · Avidity Biosciences, Inc. (2) · Richard Barohn, MD (2) · Grete Andersen, MD (1) · Centre Hospitalier Universitaire de Nice (1) · Lupin Ltd. (1) · Charite University, Berlin, Germany (1)
What is being tested here
Drugs under test in Myotonia studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| AOC 1001 | 0 | 1 | 1 |
| Lamotrigine | 0 | 1 | 1 |
| Mexiletine | 0 | 0 | 1 |
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 2, phase 3 first
- Jan 2027An Open-Label, Randomized, Cross-Over Study to Investigate the Efficacy and Safety of Mexiletine PR Compared to Mexiletine IR · Phase 3 · Lupin Ltd.
- Feb 2027A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) · Vertex Pharmaceuticals Incorporated
Who these studies take
Of the 4 Myotonia studies recruiting now, 2 (50%) set no upper age limit and 2 (50%) stop at 75 or younger. 3 (75%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 0 (0%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 1 (25%) | 1 (25%) |
| 18 | 3 (75%) | 3 (75%) |
| 30 | 3 (75%) | 3 (75%) |
| 40 | 3 (75%) | 3 (75%) |
| 50 | 3 (75%) | 3 (75%) |
| 65 | 3 (75%) | 3 (75%) |
| 75 | 2 (50%) | 2 (50%) |
| 85 | 2 (50%) | 2 (50%) |
Across all 17 studies, recruiting or not: 35% stop at 75 or younger and 24% at 65; 47% take children; 0% are for women only and 0% for men only; 6% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
What changed recently
From the registry's own updates since Oct 13, 2026.
Newly registered1
- Telerehabilitation Hand Training for Patients With Neuromuscular ConditionsNov 9, 2026NCT07926971
Studies
Recruiting first, then most recently updated. Showing 17 of 17. search them.
- Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1RecruitingNCT07486934Phase 312 countries
- Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) ExtensionRecruitingNCT077002251 country
- A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type IEnrolling by invitationNCT06926621Phase 28 countries
- Global Open-Label Extension Study of Del-desiran for the Treatment of DM1Enrolling by invitationNCT07008469Phase 310 countries
- Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy ExtensionRecruitingNCT067478842 countries
- Electromyostimulation (EMS) of Astronauts in Spaceflight (Easymotion-2)Enrolling by invitationNCT06492057Not applicable1 country
- An Open-Label, Randomized, Cross-Over Study to Investigate the Efficacy and Safety of Mexiletine PR Compared to Mexiletine IRRecruitingNCT07097701Phase 31 country
- Telerehabilitation Hand Training for Patients With Neuromuscular ConditionsCompletedNCT07926971Not applicable1 country
- Global Study of Del-desiran for the Treatment of DM1CompletedNCT06411288Phase 310 countries
- A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)Active, not recruitingNCT06185764Phase 1/210 countries
- Pilot Study for the Development of an Activity and Quality of Life Questionnaire for the Follow-up of Patients With Non-dystrophic MyotoniaCompletedNCT06136416Not applicable1 country
- MExiletine Versus Lamotrigine in Non-Dystrophic MyotoniasStatus unknownNCT05017155Phase 31 country
- Assessing Clinical Endpoints and Biomarkers in Myotonic Dystrophy Type-1 and Type 2 (ASCEND-DM)TerminatedNCT038674351 country
- Lamotrigine as Treatment of MyotoniaCompletedNCT01939561Phase 31 country
- Effectiveness of Mexiletine for Treating People With Non-Dystrophic MyotoniaCompletedNCT00832000Phase 24 countries
- Characteristics of Nondystrophic MyotoniasCompletedNCT002444133 countries
- Cardiovascular Consequences of NIV Withdrawal in Patients With Myotonic DystrophyStatus unknownNCT00745238Not applicable1 country