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Trial Match

Clinical trials test new treatments — sometimes the newest options available for a disease. Find trials that are recruiting now, near you. Open one to see its eligibility rules as simple questions to go through with your doctor.

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30 recruiting trials

for Myotonic Disorders

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 NCT06844214Phase 1, Phase 210 sites📍 Gainesville📍 Tampa📍 New York Sanofi · Tests: SAR446268 Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology NCT07136844NA1 site📍 Liège Centre Hospitalier Universitaire de Liege · Tests: Syde, Dynamometric measurements of muscle strength, The 6-minute walk test (6MWT), The 10-meter test (10MWT), The 4 sta… Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders NCT06147414n/a1 site📍 Paris Assistance Publique - Hôpitaux de Paris · Tests: Blood sample DMCRN-02-001: Assessing Pediatric Endpoints in DM1 NCT05224778n/a5 sites📍 Los Angeles📍 Fairway📍 Rochester Virginia Commonwealth University Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1 NCT07486934Phase 343 sites📍 La Jolla📍 Los Angeles📍 Palo Alto Dyne Therapeutics · Tests: zeleciment basivarsen (DYNE-101), Placebo Biomarker Development for Muscular Dystrophies NCT05019625n/a5 sites📍 Boston📍 Boston📍 Boston Massachusetts General Hospital Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers NCT07321977NA1 site📍 Paris Institut de Myologie, France · Tests: Gait analysis via video capture Characterization of Patients With Cardiomyopathy to Identify Critical Patients Candidates for Cardiac Transplantation NCT06813443n/a3 sites📍 Bologna📍 Roma📍 Florence IRCCS Azienda Ospedaliero-Universitaria di Bologna The Natural History and Muscle Fatigability of Patients With Congenital Myopathies. NCT06157268n/a2 sites📍 Nijmegen📍 Utrecht Radboud University Medical Center · Tests: Natural history and non therapeutical therapy Development of Quantitative Muscle Imaging as a Biomarker of Disease Endpoints in Myotonic Dystrophy NCT07362875n/a1 site📍 Winston-Salem Wake Forest University Health Sciences Myotonic Dystrophy Family Registry NCT02398786n/a1 site📍 Oakland Myotonic Dystrophy Foundation · Tests: Patient-entered data A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 NCT06667453Phase 212 sites📍 Calgary📍 Ottawa📍 Chicoutimi PepGen Inc · Tests: PGN-EDODM1, Placebo Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases NCT06833489NA1 site📍 Marseille Assistance Publique Hopitaux De Marseille · Tests: ARN extraction from muscle biopsies Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension NCT07700225n/a1 site📍 Richmond Virginia Commonwealth University Remote Assessments and Genetic Determinants of Congenital and Childhood Myotonic Dystrophy NCT07630389n/a1 site📍 Rochester University of Rochester Personalized Training for People With Rare Neuromuscular Disorders NCT06708468NA5 sites📍 Bergen📍 Molde📍 Namsos Oslo University Hospital · Tests: Exercise, Usual Care A Natural History Study of RYR1-Related Disorders NCT06287762n/a1 site📍 Bethesda National Institutes of Health Clinical Center (CC) Muscle Health Measurements Using Electrical Impedance Myography NCT07502989n/a1 site📍 Boston Beth Israel Deaconess Medical Center · Tests: Electrical Impedance Myography Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease) NCT05532813Phase 31 site📍 Garches Assistance Publique - Hôpitaux de Paris · Tests: Treatment taken Prospective, Long Term, Observational Study (Patient Registry) of Paediatric Myotonic Disorders NCT07154654n/a1 site📍 Clermont-Ferrand Lupin Ltd. · Tests: Mexiletine Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study NCT05982119NA8 sites📍 Liège📍 Prague📍 Cairo Centre Hospitalier Universitaire de Liege · Tests: ActiMyo/Syde Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO) NCT07608432Phase 31 site📍 Hillsborough Dyne Therapeutics · Tests: Zeleciment Rostudirsen (DYNE-251), Placebo Pelvic Floor Muscle Training for Women with Myotonic Dystrophy NCT06316778NA1 site📍 Jonquière Université de Sherbrooke · Tests: Pelvic floor muscle training Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry NCT00082108n/a1 site📍 Rochester University of Rochester The Spanish National Registry for Myotonic Dystrophy Type 1 NCT07385443n/a8 sites📍 Multiple Locations📍 Multiple Locations📍 Multiple Locations Fundació Institut Germans Trias i Pujol · Tests: Patient Registry Evaluation of the Role of miR-1 in the Pathogenesis and as a Biomarker in Muscular Dystrophies and Congenital Myopathies NCT07415837NA1 site📍 Clermont-Ferrand University Hospital, Clermont-Ferrand · Tests: dosage of blood biomarker miR1 Study of ATX-01 in Participants With DM1 NCT06300307Phase 1, Phase 212 sites📍 Los Angeles📍 Gainesville📍 Iowa City ARTHEx Biotech S.L. · Tests: ATX-01, Placebo Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy NCT05004129Phase 2, Phase 314 sites📍 Little Rock📍 Los Angeles📍 Palo Alto AMO Pharma Limited · Tests: Tideglusib Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) NCT03981575n/a17 sites📍 La Jolla📍 Los Angeles📍 Denver Virginia Commonwealth University An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE) NCT07220603Phase 23 sites📍 Calgary📍 Ottawa📍 Saguenay PepGen Inc · Tests: PGN-EDODM1
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