Myotonic Dystrophy 1 Clinical Trials

11 recruiting

Myotonic Dystrophy 1 Trials at a Glance

11 actively recruiting trials for myotonic dystrophy 1 are listed on ClinicalTrialsFinder across 6 cities in 19 countries. The largest study group is Phase 2 with 4 trials, with the heaviest enrollment activity in Gainesville, Liège, and Calgary. Lead sponsors running myotonic dystrophy 1 studies include Centre Hospitalier Universitaire de Liege, PepGen Inc, and Assistance Publique - Hôpitaux de Paris.

Browse myotonic dystrophy 1 trials by phase

Treatments under study

About Myotonic Dystrophy 1 Clinical Trials

Looking for clinical trials for Myotonic Dystrophy 1? There are currently 11 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 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 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 2

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

Myotonic Dystrophy 1
PepGen Inc24 enrolled8 locationsNCT06667453
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
Phase 1Phase 2

Study of ATX-01 in Participants With DM1

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

Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease)

MetforminMyotonic Dystrophy 1Steinert's Disease
Assistance Publique - Hôpitaux de Paris142 enrolled1 locationNCT05532813
Recruiting
Phase 1Phase 2

Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy

Myotonic Dystrophy 1
Arrowhead Pharmaceuticals78 enrolled11 locationsNCT06138743
Recruiting
Not Applicable

Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology

Myasthenia GravisHuntington DiseaseNeuromuscular Diseases+9 more
Centre Hospitalier Universitaire de Liege300 enrolled1 locationNCT07136844
Recruiting

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

Myotonic Dystrophy 1DM1
Virginia Commonwealth University700 enrolled17 locationsNCT03981575
Recruiting
Not Applicable

Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study

Duchenne Muscular DystrophyFascioscapulohumeral Muscular DystrophyCentronuclear Myopathy+3 more
Centre Hospitalier Universitaire de Liege300 enrolled8 locationsNCT05982119
Recruiting

Myotonic Dystrophy Family Registry

Myotonic DystrophyMyotonic Dystrophy 1Dystrophia Myotonica+13 more
Myotonic Dystrophy Foundation3,500 enrolled1 locationNCT02398786
Recruiting

Myotonic Dystrophy Type 1 Congenital and Juvenile Form: From Diagnosis to Rehabilitation [MDCJ-NeuBeRe]

Myotonic Dystrophy 1
IRCCS Eugenio Medea30 enrolled1 locationNCT06378216