Myotonic Dystrophy clinical trials
The registry lists 126 studies for Myotonic Dystrophy across more than 10 countries. 50 are recruiting or about to start. 103 of them are mainly about Myotonic Dystrophy (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-10.
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Status
Who runs them
University of Rochester (8) · Lupin Ltd. (6) · Virginia Commonwealth University (6) · Massachusetts General Hospital (5) · Avidity Biosciences, Inc. (5) · Institut de Myologie, France (5) · LMU Klinikum (5) · AMO Pharma Limited (4)
What is being tested here
Drugs under test in Myotonic Dystrophy studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
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.
The 10 nearest, phase 3 first
- Nov 2026Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease) · Phase 3 · Assistance Publique - Hôpitaux de Paris
- Dec 2026Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy · Phase 3 · AMO Pharma Limited
- Jun 2027The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2 · Phase 3 · Lupin Ltd.
- Dec 2026Extracellular RNA Biomarkers of Myotonic Dystrophy · Massachusetts General Hospital
- Dec 2026DMCRN-02-001: Assessing Pediatric Endpoints in DM1 · Virginia Commonwealth University
- Dec 2026Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) · Virginia Commonwealth University
- Dec 2026Pelvic Floor Muscle Training for Women with Myotonic Dystrophy · Université de Sherbrooke
- Dec 2026Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy · Sarepta Therapeutics, Inc.
- Dec 2026The Spanish National Registry for Myotonic Dystrophy Type 1 · Fundació Institut Germans Trias i Pujol
- Dec 2026Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers · Institut de Myologie, France
Who these studies take
Of the 35 Myotonic Dystrophy studies recruiting now, 15 (43%) set no upper age limit and 18 (51%) stop at 75 or younger. 22 (63%) take children under 18. 2 (6%) are for women only and 2 (6%) for men only; 8 (23%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 14 (40%) | 16 (46%) |
| 18 | 30 (86%) | 29 (83%) |
| 30 | 30 (86%) | 28 (80%) |
| 40 | 29 (83%) | 27 (77%) |
| 50 | 28 (80%) | 26 (74%) |
| 65 | 25 (71%) | 23 (66%) |
| 75 | 17 (49%) | 15 (43%) |
| 85 | 16 (46%) | 14 (40%) |
Across all 126 studies, recruiting or not: 50% stop at 75 or younger and 40% at 65; 34% take children; 3% are for women only and 2% for men only; 26% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
How long Myotonic Dystrophy studies take
Of 36 observational studies under Myotonic Dystrophy, half reached primary completion within 60 months of starting, counting those still running.
| Start to primary completion | Every sponsor | Industry | Non-industry |
|---|---|---|---|
| Interventional, no phase | 23 mo12–57 · 25 studies | — | — |
| Observational | 60 mo35–… · 36 studies | — | — |
Each figure is the month by which half the studies had reached primary completion, with the range in which the middle half did. Studies still running count until their last registry update; studies that stopped early are left out. Phase 1 includes early phase 1; observational means prospective or not stated, as retrospective and cross-sectional studies date a window of data rather than a study. How these figures are worked out
What changed recently
From the registry's own updates since Oct 13, 2026.
Newly registered2
- Telerehabilitation Hand Training for Patients With Neuromuscular ConditionsNov 9, 2026NCT07926971
- Metabolomic Biomarkers of Myotonic DystrophyNov 3, 2026NCT07912983
Studies
Recruiting first, then most recently updated. Showing 50 of 126. Browse all 126 → or search them.
- A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 SkippingRecruitingNCT07587242Phase 310 countries
- Calcium Channel Blocker in Myotonic Dystrophy Type 1Not yet recruitingNCT07075965Phase 1
- Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1RecruitingNCT07486934Phase 312 countries
- Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic VolunteersRecruitingNCT07321977Not applicable1 country
- Metabolomic Biomarkers of Myotonic DystrophyRecruitingNCT079129831 country
- DMCRN-02-001: Assessing Pediatric Endpoints in DM1RecruitingNCT052247782 countries
- Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)RecruitingNCT039815757 countries
- The Spanish National Registry for Myotonic Dystrophy Type 1RecruitingNCT073854431 country
- Biomarker Development for Muscular DystrophiesRecruitingNCT050196251 country
- Cerebrospinal Fluid Biomarkers of Myotonic DystrophyRecruitingNCT060756931 country
- Development of Non-Invasive Prenatal Diagnosis for Single Gene DisordersRecruitingNCT061474141 country
- 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
- Investigating Exercise in Myotonic Dystrophy Type 2 (DM2)RecruitingNCT06716931Not applicable1 country
- The United Kingdom National Registry for Myotonic DystrophyRecruitingNCT040033631 country
- Study of SRP-1003 in Participants With Type 1 Myotonic DystrophyEnrolling by invitationNCT06138743Phase 1/211 countries
- The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2RecruitingNCT06523400Phase 36 countries
- A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1RecruitingNCT06844214Phase 1/26 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
- A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1RecruitingNCT06667453Phase 24 countries
- An Ambispective Natural History Study in Myotonic Dystrophy Patients Linking Retrospective Data Captured From the DM-Scope Registry With a Prospective 24-month Follow-up PeriodNot yet recruitingNCT077324391 country
- Brain Structure and Clinical Endpoints in Myotonic Dystrophy Type 2Enrolling by invitationNCT058544331 country
- Personalized Training for People With Rare Neuromuscular DisordersRecruitingNCT06708468Not applicable1 country
- Remote Assessments and Genetic Determinants of Congenital and Childhood Myotonic DystrophyRecruitingNCT076303891 country
- Chinese Multicenter Clinical Outcome Cohort Study of Myotonic Dystrophy Type 1 (C-DMCOS-DM1)Enrolling by invitationNCT061019401 country
- Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)RecruitingNCT07608432Phase 31 country
- Remote Assessments and Genetic Determinants of Myotonic DystrophyRecruitingNCT075053421 country
- An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE)RecruitingNCT07220603Phase 21 country
- Evaluation of the Role of miR-1 in the Pathogenesis and as a Biomarker in Muscular Dystrophies and Congenital MyopathiesRecruitingNCT07415837Not applicable1 country
- Study of ATX-01 in Participants With DM1RecruitingNCT06300307Phase 1/27 countries
- An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2 Who Have Completed MEX-DM-302 Study.Enrolling by invitationNCT06549400Phase 36 countries
- Development of Quantitative Muscle Imaging as a Biomarker of Disease Endpoints in Myotonic DystrophyRecruitingNCT073628751 country
- Derivation of Human Induced Pluripotent Stem (iPS) Cells to Heritable Cardiac ArrhythmiasEnrolling by invitationNCT024134501 country
- Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease)RecruitingNCT05532813Phase 31 country
- Long-Term Development of Muscular Dystrophy Outcome AssessmentsRecruitingNCT059896201 country
- Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy RegistryRecruitingNCT000821081 country
- Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular DisordersEnrolling by invitationNCT066056121 country
- Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic PathologyRecruitingNCT07136844Not applicable1 country
- Tideglusib: Expanded Access Use in Congenital Myotonic DystrophyAvailableNCT07119775
- The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.Enrolling by invitationNCT07072676Not applicable1 country
- Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic DystrophyRecruitingNCT05004129Phase 2/34 countries
- Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next StudyRecruitingNCT05982119Not applicable7 countries
- A Registered Observational Cohort Study of Myotonic Dystrophy Type 1Enrolling by invitationNCT069790241 country
- Pelvic Floor Muscle Training for Women with Myotonic DystrophyRecruitingNCT06316778Not applicable1 country
- Characterization of Patients With Cardiomyopathy to Identify Critical Patients Candidates for Cardiac TransplantationRecruitingNCT068134431 country
- Myotonic Dystrophy Family RegistryRecruitingNCT023987861 country
- Wheelchair Skills Training for People with ARSACS and DM1Not yet recruitingNCT06596850Not applicable1 country
- Myotonic Dystrophy Type 1 Congenital and Juvenile Form: From Diagnosis to Rehabilitation [MDCJ-NeuBeRe]RecruitingNCT063782161 country
- Home-based Training and Supplementation in DM1 PatientsNot yet recruitingNCT05848830Phase 31 country