Myotonic Dystrophy Clinical Trials

35 recruitingLast updated: June 18, 2026

There are 35 actively recruiting myotonic dystrophy clinical trials across 24 countries. Studies span Phase 2, Not Applicable, Phase 1, Phase 3. Top locations include Richmond, Virginia, United States, Rochester, New York, United States, Paris, France. Updated daily from ClinicalTrials.gov.


Myotonic Dystrophy Trials at a Glance

35 actively recruiting trials for myotonic dystrophy are listed on ClinicalTrialsFinder across 6 cities in 24 countries. The largest study group is Phase 2 with 8 trials, with the heaviest enrollment activity in Richmond, Rochester, and Paris. Lead sponsors running myotonic dystrophy studies include Massachusetts General Hospital, Dyne Therapeutics, and University of Rochester.

Browse myotonic dystrophy trials by phase

Treatments under study

About Myotonic Dystrophy Clinical Trials

Looking for clinical trials for Myotonic Dystrophy? There are currently 12 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new Myotonic Dystrophy trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

Frequently Asked Questions

Common questions about Myotonic Dystrophy clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 120 of 35 trials

Recruiting

Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)

Myotonic Dystrophy 1DM1
Virginia Commonwealth University700 enrolled17 locationsNCT03981575
Recruiting

DMCRN-02-001: Assessing Pediatric Endpoints in DM1

Congenital Myotonic DystrophyCDM
Virginia Commonwealth University50 enrolled5 locationsNCT05224778
Recruiting
Phase 1Phase 2

A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)

Myotonic Dystrophy Type 1 (DM1)
Vertex Pharmaceuticals Incorporated52 enrolled26 locationsNCT06185764
Recruiting

Remote Assessments and Genetic Determinants of Congenital and Childhood Myotonic Dystrophy

Myotonic Dystrophy Type 1Congenital DM1Juvenile DM1+1 more
University of Rochester100 enrolled1 locationNCT07630389
Recruiting
Phase 1Phase 2

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 1

Myotonic Dystrophy
Sanofi32 enrolled9 locationsNCT06844214
Recruiting
Phase 3

Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1

Myotonic DystrophyDM1Steinert Disease+2 more
Dyne Therapeutics150 enrolled6 locationsNCT07486934
Recruiting
Phase 3

Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)

Muscular Dystrophy, DuchenneDMDNeuromuscular Diseases (NMD)+10 more
Dyne Therapeutics90 enrolled1 locationNCT07608432
Recruiting
Not Applicable

Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers

Myotonic DystrophyMuscular DystrophyCharcot-Marie-Tooth+1 more
Institut de Myologie, France30 enrolled1 locationNCT07321977
Recruiting
Phase 1Phase 2

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1

Myotonic Dystrophy Type 1 (DM1)
Dyne Therapeutics116 enrolled20 locationsNCT05481879
Recruiting
Phase 2

A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1

Myotonic Dystrophy 1
PepGen Inc24 enrolled8 locationsNCT06667453
Recruiting

Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders

Cystic FibrosisHuntington DiseaseSickle Cell Disease+12 more
Assistance Publique - Hôpitaux de Paris550 enrolled1 locationNCT06147414
Recruiting
Not Applicable

Investigating Exercise in Myotonic Dystrophy Type 2 (DM2)

Myotonic Dystrophy 2
Massachusetts General Hospital24 enrolled1 locationNCT06716931
Recruiting
Phase 2

An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE)

Myotonic Dystrophy 1
PepGen Inc48 enrolled3 locationsNCT07220603
Recruiting

Remote Assessments and Genetic Determinants of Myotonic Dystrophy

DM1Myotonic Dystrophy Type 1 (DM1)
University of Rochester1,000 enrolled1 locationNCT07505342
Recruiting
Not Applicable

Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1)

Myotonic Dystrophy Type 1Myotonic Dystrophy, CongenitalMyotonic Dystrophy Type 1 (DM1)
Hanns Lochmuller13 enrolled1 locationNCT06809049
Recruiting
Phase 1Phase 2

Study of ATX-01 in Participants With DM1

Myotonic Dystrophy 1
ARTHEx Biotech S.L.56 enrolled12 locationsNCT06300307
Recruiting
Phase 3

The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2

Myotonic Dystrophy
Lupin Ltd.176 enrolled7 locationsNCT06523400
Recruiting

The Spanish National Registry for Myotonic Dystrophy Type 1

Myotonic Dystrophy 1Myotonic Dystrophy Type 1DM1+2 more
Fundació Institut Germans Trias i Pujol3,000 enrolled8 locationsNCT07385443
Recruiting

Brain Structure and Clinical Endpoints in Myotonic Dystrophy Type 2

Myotonic Dystrophy Type 1Myotonic Dystrophy Type 2
Wake Forest University Health Sciences100 enrolled1 locationNCT05854433
Recruiting

Development of Quantitative Muscle Imaging as a Biomarker of Disease Endpoints in Myotonic Dystrophy

Myotonic Dystrophy
Wake Forest University Health Sciences75 enrolled1 locationNCT07362875