| Recruiting | Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy NCT07486934 | Dyne Therapeutics | Phase 3 |
| Recruiting | Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuro NCT07321977 | Institut de Myologie, France | N/A |
| Enrolling By Invitation | An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Once Daily Mexiletine PR in Pat NCT06549400 | Lupin Ltd. | Phase 3 |
| Enrolling By Invitation | Global Open-Label Extension Study of Del-desiran for the Treatment of DM1 NCT07008469 | Avidity Biosciences, Inc. | Phase 3 |
| Recruiting | A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-m NCT06844214 | Sanofi | Phase 1 / Phase 2 |
| Recruiting | Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension NCT06747884 | Virginia Commonwealth University | — |
| Recruiting | Development of Quantitative Muscle Imaging as a Biomarker of Disease Endpoints in Myotonic Dystrophy NCT07362875 | Wake Forest University Health Sciences | — |
| Recruiting | The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2 NCT06523400 | Lupin Ltd. | Phase 3 |
| Recruiting | Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders NCT06147414 | Assistance Publique - Hôpitaux de Paris | — |
| Enrolling By Invitation | Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disor NCT06605612 | LMU Klinikum | — |
| Active Not Recruiting | Global Study of Del-desiran for the Treatment of DM1 NCT06411288 | Avidity Biosciences, Inc. | Phase 3 |
| Completed | The Risk of Falls Index for Patients With Neuromuscular Disorders NCT05890833 | LMU Klinikum | — |
| Completed | Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients NCT05479981 | Avidity Biosciences, Inc. | Phase 2 |
| Completed | Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients NCT05027269 | Avidity Biosciences, Inc. | Phase 1 / Phase 2 |
| Completed | Open Label Study in Adolescents and Children With Myotonic Disorders NCT04624750 | Lupin Ltd. | Phase 3 |
| Active Not Recruiting | An Observational Study in Adult Patients With Non-dystrophic Myotonic Disorders NCT04616807 | Lupin Ltd. | — |
| Recruiting | Extracellular RNA Biomarkers of Myotonic Dystrophy NCT05020002 | Massachusetts General Hospital | — |
| Completed | Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1 NCT03959189 | Expansion Therapeutics, Inc. | Phase 1 |
| Recruiting | Biomarker Development for Muscular Dystrophies NCT05019625 | Massachusetts General Hospital | — |
| Terminated | European Home Mechanical Ventilation Registry NCT02315339 | ResMed | — |
| Completed | Sleep Breathing Disorders, a Main Trigger for Cardiac ARythmias in Type I Myotonic Dystrophy ? NCT02375087 | University Hospital, Grenoble | — |
| Completed | At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurolo NCT01931644 | Sanguine Biosciences | — |
| Recruiting | The United Kingdom National Registry for Myotonic Dystrophy NCT04003363 | Newcastle University | — |
| Recruiting | Myotonic Dystrophy Family Registry NCT02398786 | Myotonic Dystrophy Foundation | — |
| Completed | Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1 NCT01406873 | University of Rochester | Phase 2 |
| Completed | DM1 Heart Registry - DM1 Respiratory Registry NCT01136330 | Institut de Myologie, France | — |
| Completed | Postural Spirometry Changes in Ambulatory Myotonic Dystrophy Patients NCT01242007 | University of Nancy | N/A |
| Completed | Effects of SomatoKine (Iplex)Recombinant Human Insulin-like Growth Factor-1/Recombinant Human Insulin-like Gro NCT00233519 | University of Rochester | Phase 1 / Phase 2 |
| Completed | Efficacy and Safety of DHEA for Myotonic Dystrophy NCT00167609 | University of Versailles | Phase 2 / Phase 3 |
| Completed | RAMYD Study - Evaluation of Arrhythmic Risk in Myotonic Dystrophy NCT00127582 | Catholic University of the Sacred Heart | N/A |
| Recruiting | Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry NCT00082108 | University of Rochester | — |