Myotonic dystrophy trials in United States

In United States 23 Also abroad 45

23 trials · From ClinicalTrials.gov, checked 15 November 2026. Ask the study team whether a trial fits you.

Phase 3Taking people
Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
Where
Atlanta, Baltimore, Columbia, Gainesville, Hillsborough, Houston, Indianapolis, La Jolla, Los Angeles, Palo Alto, Richmond, Rochester, Salt Lake City, San Antonio
Age
16 years and over
People
150 worldwide
Run by
Dyne Therapeutics
See details and how to joinNCT07486934 · EU 2025-522957-20-00
No phaseTaking people
Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)
Where
Columbus, Denver, Gainesville, Houston, Iowa City, Kansas City, La Jolla, Los Angeles, Richmond, Rochester
Age
18 years to 70 years
People
700 worldwide
Run by
Virginia Commonwealth University
See details and how to joinNCT03981575
No phaseTaking people
DMCRN-02-001: Assessing Pediatric Endpoints in DM1
Where
Fairway, Los Angeles, Richmond, Rochester
Age
Up to 59 months
People
50 worldwide
Run by
Virginia Commonwealth University
See details and how to joinNCT05224778
No phaseTaking people
Metabolomic Biomarkers of Myotonic Dystrophy
Where
Boston
Age
14 years and over
People
64
Run by
Massachusetts General Hospital
See details and how to joinNCT07912983
No phaseTaking people
Biomarker Development for Muscular Dystrophies
Where
Boston, Pittsburgh, Winston-Salem
Age
5 years and over
People
465
Run by
Massachusetts General Hospital
See details and how to joinNCT05019625
No phaseTaking people
Cerebrospinal Fluid Biomarkers of Myotonic Dystrophy
Where
Boston
Age
18 years and over
People
88
Run by
Massachusetts General Hospital
See details and how to joinNCT06075693
No phaseTaking people
Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension
Where
Richmond
Age
18 years to 70 years
People
1,000
Run by
Virginia Commonwealth University
See details and how to joinNCT07700225
Phase 2Enrolling by invitation
A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I
Where
Richmond, St Louis, Winston-Salem
Age
18 years and over
People
44 worldwide
Run by
Vertex Pharmaceuticals Incorporated
See details and how to joinNCT06926621 · EU 2024-517983-47-00
No phaseTaking people
Investigating Exercise in Myotonic Dystrophy Type 2 (DM2)
Where
Boston
Age
18 years to 70 years
People
24
Run by
Massachusetts General Hospital
See details and how to joinNCT06716931
Phase 1/2Taking people
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
Where
Gainesville, New York, Philadelphia, Richmond, Tampa
Age
10 years to 55 years
People
32 worldwide
Run by
Sanofi
See details and how to joinNCT06844214
Phase 3Enrolling by invitation
Global Open-Label Extension Study of Del-desiran for the Treatment of DM1
Where
Baltimore, Boston, Cincinnati, Columbus, Denver, Durham, Gainesville, Houston, Indianapolis, Kansas City, Los Angeles, Minneapolis, Philadelphia, Richmond, Rochester, Seattle, Stanford, Tampa, Winston-Salem
Age
16 years and over
People
199 worldwide
Run by
Avidity Biosciences, Inc.
See details and how to joinNCT07008469
No phaseTaking people
Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension
Where
Kansas City, Little Rock, Richmond
Age
3 years to 17 years
People
200 worldwide
Run by
Virginia Commonwealth University
See details and how to joinNCT06747884
No phaseEnrolling by invitation
Brain Structure and Clinical Endpoints in Myotonic Dystrophy Type 2
Where
Winston-Salem
Age
30 years to 65 years
People
100
Run by
Wake Forest University Health Sciences
See details and how to joinNCT05854433
No phaseTaking people
Remote Assessments and Genetic Determinants of Congenital and Childhood Myotonic Dystrophy
Where
Rochester
Age
0 years to 17 years
People
100
Run by
University of Rochester
See details and how to joinNCT07630389
Phase 3Taking people
Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
Where
Hillsborough
Age
4 years to 18 years
People
90
Run by
Dyne Therapeutics
See details and how to joinNCT07608432 · EU 2025-524096-23-00
No phaseTaking people
Remote Assessments and Genetic Determinants of Myotonic Dystrophy
Where
Rochester
Age
18 years to 88 years
People
1,000
Run by
University of Rochester
See details and how to joinNCT07505342
Phase 1/2Taking people
Study of ATX-01 in Participants With DM1
Where
Fairway, Gainesville, Iowa City, Los Angeles, Richmond
Age
18 years to 64 years
People
56 worldwide
Run by
ARTHEx Biotech S.L.
See details and how to joinNCT06300307
No phaseTaking people
Development of Quantitative Muscle Imaging as a Biomarker of Disease Endpoints in Myotonic Dystrophy
Where
Winston-Salem
Age
18 years to 65 years
People
75
Run by
Wake Forest University Health Sciences
See details and how to joinNCT07362875
No phaseEnrolling by invitation
Derivation of Human Induced Pluripotent Stem (iPS) Cells to Heritable Cardiac Arrhythmias
Where
Baltimore
Age
18 years to 85 years
People
100
Run by
Johns Hopkins University
See details and how to joinNCT02413450
No phaseTaking people
Long-Term Development of Muscular Dystrophy Outcome Assessments
Where
Richmond
Age
6 years to 50 years
People
1,000
Run by
Virginia Commonwealth University
See details and how to joinNCT05989620
No phaseTaking people
Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry
Where
Rochester
People
3,000
Run by
University of Rochester
See details and how to joinNCT00082108
Phase 2/3Taking people
Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy
Where
Chicago, Iowa City, Little Rock, Los Angeles, Norfolk, Palo Alto, Pittsburgh, Richmond, Rochester, Salt Lake City
Age
6 years to 45 years
People
76 worldwide
Run by
AMO Pharma Limited
See details and how to joinNCT05004129
No phaseTaking people
Myotonic Dystrophy Family Registry
Where
Oakland
People
3,500
Run by
Myotonic Dystrophy Foundation
See details and how to joinNCT02398786