Myotonic Dystrophy Type 1 Clinical Trials

11 recruitingLast updated: May 13, 2026

There are 11 actively recruiting myotonic dystrophy type 1 clinical trials across 13 countries. Studies span Not Applicable, Phase 1, Phase 2, Phase 3. Top locations include Rochester, New York, United States, Richmond, Virginia, United States, London, United Kingdom. Updated daily from ClinicalTrials.gov.


Myotonic Dystrophy Type 1 Trials at a Glance

11 actively recruiting trials for myotonic dystrophy type 1 are listed on ClinicalTrialsFinder across 6 cities in 13 countries. The largest study group is Not Applicable with 3 trials, with the heaviest enrollment activity in Rochester, Richmond, and London. Lead sponsors running myotonic dystrophy type 1 studies include Dyne Therapeutics, University of Rochester, and Hanns Lochmuller.

Browse myotonic dystrophy type 1 trials by phase

Treatments under study

About Myotonic Dystrophy Type 1 Clinical Trials

Looking for clinical trials for Myotonic Dystrophy Type 1? There are currently 6 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 Type 1 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 Type 1 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 111 of 11 trials

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 enrolled5 locationsNCT07486934
Recruiting

Remote Assessments and Genetic Determinants of Myotonic Dystrophy

DM1Myotonic Dystrophy Type 1 (DM1)
University of Rochester1,000 enrolled1 locationNCT07505342
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 enrolled17 locationsNCT05481879
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

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

Myotonic Dystrophy Type 1 (DM1)
Vertex Pharmaceuticals Incorporated44 enrolled26 locationsNCT06185764
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

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

Cerebrospinal Fluid Biomarkers of Myotonic Dystrophy

Myotonic Dystrophy Type 1
Massachusetts General Hospital88 enrolled1 locationNCT06075693
Recruiting

Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry

facioscapulohumeral muscular dystrophyMyotonic DystrophyMuscular Dystrophy+6 more
University of Rochester3,000 enrolled1 locationNCT00082108
Recruiting
Not Applicable

Personalized Training for People With Rare Neuromuscular Disorders

facioscapulohumeral muscular dystrophyNeuromuscular Diseases (NMD)Charcot Marie Tooth Disease (CMT)+1 more
Oslo University Hospital120 enrolled5 locationsNCT06708468
Recruiting
Not Applicable

Pelvic Floor Muscle Training for Women with Myotonic Dystrophy

Urinary IncontinenceMyotonic Dystrophy Type 1
Université de Sherbrooke12 enrolled1 locationNCT06316778