myotonic dystrophy
myotonic dystrophy
Explore linked clinical trials. Standard reference: MONDO:001610752 results · Page 1 of 3
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Investigating Exercise in Myotonic Dystrophy Type 2 (DM2) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, Myotonic Dystrophy 2, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Recruiting | Not applicable | 2026-10-08 | NCT06716931 | |
| The United Kingdom National Registry for Myotonic Dystrophy | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Recruiting | Not stated | 2026-10-06 | NCT04003363 | |
| Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, Myotonia, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic dystrophy type 1, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Recruiting | Phase 3 | 2026-10-05 | NCT07486934 | |
| Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Enrolling by invitation | Phase 1 / Phase 2 | 2026-10-05 | NCT06138743 | |
| A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Infant, Newborn, Diseases, Muscular Disorders, Atrophic, X-linked disease, congenital, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Recruiting | Phase 3 | 2026-09-29 | NCT07587242 | |
| The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Recruiting | Phase 3 | 2026-09-24 | NCT06523400 | |
| Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Active, not recruiting | Phase 1 / Phase 2 | 2026-09-23 | NCT05481879 | |
| 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 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Recruiting | Phase 1 / Phase 2 | 2026-09-21 | NCT06844214 | |
| Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, Myotonia, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic dystrophy type 1, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Recruiting | Not stated | 2026-09-04 | NCT07700225 | |
| A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, Myotonia, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Active, not recruiting | Phase 1 / Phase 2 | 2026-08-20 | NCT06185764 | |
| Global Open-Label Extension Study of Del-desiran for the Treatment of DM1 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, Myotonia, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic dystrophy type 1, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Enrolling by invitation | Phase 3 | 2026-08-19 | NCT07008469 | |
| Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, Myotonia, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Recruiting | Not stated | 2026-08-11 | NCT06747884 | |
| A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, Myotonia, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Enrolling by invitation | Phase 2 | 2026-08-10 | NCT06926621 | |
| A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Recruiting | Phase 2 | 2026-07-29 | NCT06667453 | |
| An Ambispective Natural History Study in Myotonic Dystrophy Patients Linking Retrospective Data Captured From the DM-Scope Registry With a Prospective 24-month Follow-up Period | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, Myotonic Dystrophy 2, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Not yet recruiting | Not stated | 2026-07-28 | NCT07732439 | |
| Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Muscular Disorders, Atrophic, Myotonic Dystrophy, Congenital, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rare Diseases, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic dystrophy type 1, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Active, not recruiting | Not applicable | 2026-07-28 | NCT06809049 | |
| Brain Structure and Clinical Endpoints in Myotonic Dystrophy Type 2 | Cognition Disorders, Cognitive Dysfunction, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, cognitive disorder, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic dystrophy type 1, myotonic dystrophy type 2, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease, psychiatric disorder | Enrolling by invitation | Not stated | 2026-07-14 | NCT05854433 | |
| Personalized Training for People With Rare Neuromuscular Disorders | Charcot-Marie-Tooth disease, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, central nervous system malformation, facioscapulohumeral muscular dystrophy, hereditary disease, hereditary motor and sensory neuropathy, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease, peripheral nervous system disorder, polyneuropathy | Recruiting | Not applicable | 2026-06-18 | NCT06708468 | |
| DMCRN-02-001: Assessing Pediatric Endpoints in DM1 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Recruiting | Not stated | 2026-06-10 | NCT05224778 | |
| Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic dystrophy type 1, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Recruiting | Not stated | 2026-06-10 | NCT03981575 | |
| Remote Assessments and Genetic Determinants of Congenital and Childhood Myotonic Dystrophy | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic dystrophy type 1, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Recruiting | Not stated | 2026-06-05 | NCT07630389 | |
| Chinese Multicenter Clinical Outcome Cohort Study of Myotonic Dystrophy Type 1 (C-DMCOS-DM1) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Enrolling by invitation | Not stated | 2026-06-02 | NCT06101940 | |
| Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne and Becker Muscular Dystrophy, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease | Recruiting | Phase 3 | 2026-05-27 | NCT07608432 | |
| Myotonic Dystrophy - Vascular and Cognition | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Insulin Resistance, Muscular Disorders, Atrophic, Nutritional and Metabolic Diseases, diabetes mellitus, endocrine system disorder, glucose metabolism disease, glucose-galactose malabsorption, hereditary disease, hyperinsulinism, inherited neurodegenerative disorder, metabolic disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease, tauopathy | Active, not recruiting | Not stated | 2026-05-22 | NCT04656210 | |
| Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers | Charcot-Marie-Tooth disease, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, Pathologic Processes, Pathological Conditions, Signs and Symptoms, central nervous system disorder, central nervous system malformation, disease, hereditary disease, hereditary motor and sensory neuropathy, inherited neurodegenerative disorder, motor neuron disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular disease, peripheral nervous system disorder, polyneuropathy, spinal cord disorder, spinal muscular atrophy | Recruiting | Not applicable | 2026-05-19 | NCT07321977 |