{"disease":{"id":"myotonic-dystrophy","name":"myotonic dystrophy"},"drugs":{"marketed":[],"pipeline":[],"offLabel":[],"totalMarketed":0,"totalPipeline":0},"trials":{"data":[{"nct_id":"NCT07385443","title":"The Spanish National Registry for Myotonic Dystrophy Type 1","phase":"","overall_status":"RECRUITING","enrollment_count":3000,"lead_sponsor_name":"Fundació Institut Germans Trias i Pujol","has_results":false},{"nct_id":"NCT07505342","title":"Remote Assessments and Genetic Determinants of Myotonic Dystrophy","phase":"","overall_status":"RECRUITING","enrollment_count":1000,"lead_sponsor_name":"University of Rochester","has_results":false},{"nct_id":"NCT03981575","title":"Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)","phase":"","overall_status":"RECRUITING","enrollment_count":700,"lead_sponsor_name":"Virginia Commonwealth University","has_results":false},{"nct_id":"NCT06101940","title":"A Multicenter Phenotype-Genotype Analysis of DM1 Patients in China","phase":"","overall_status":"ENROLLING_BY_INVITATION","enrollment_count":500,"lead_sponsor_name":"Huashan Hospital","has_results":false},{"nct_id":"NCT02118779","title":"Observational Prolonged Trial in Myotonic Dystrophy Type 1","phase":"NA","overall_status":"COMPLETED","enrollment_count":255,"lead_sponsor_name":"Radboud University Medical Center","has_results":false},{"nct_id":"NCT05865483","title":"Profile of Dysphagia in Myotonic Dystrophy Type 1 (DM1)","phase":"","overall_status":"COMPLETED","enrollment_count":177,"lead_sponsor_name":"University College, London","has_results":false},{"nct_id":"NCT06549400","title":"An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2 Who Have Completed MEX-DM-302 Study.","phase":"PHASE3","overall_status":"ENROLLING_BY_INVITATION","enrollment_count":176,"lead_sponsor_name":"Lupin Ltd.","has_results":false},{"nct_id":"NCT06523400","title":"The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2","phase":"PHASE3","overall_status":"RECRUITING","enrollment_count":176,"lead_sponsor_name":"Lupin Ltd.","has_results":false},{"nct_id":"NCT03424460","title":"Venous Thromboembolism in Myotonic Dystrophy Type 1","phase":"NA","overall_status":"COMPLETED","enrollment_count":130,"lead_sponsor_name":"Assistance Publique - Hôpitaux de Paris","has_results":false},{"nct_id":"NCT06089018","title":"Observational Study of Digital Biomarkers of Myotonia and Gait in Adults and Children With Myotonic Dystrophy","phase":"","overall_status":"UNKNOWN","enrollment_count":125,"lead_sponsor_name":"Stanford University","has_results":false},{"nct_id":"NCT03764150","title":"Factors Associated With Hypoventilation in the Myotonic Dystrophy, Progressive Profile Over 5 Years","phase":"","overall_status":"COMPLETED","enrollment_count":113,"lead_sponsor_name":"University Hospital, Lille","has_results":false},{"nct_id":"NCT06605612","title":"Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders","phase":"","overall_status":"ENROLLING_BY_INVITATION","enrollment_count":108,"lead_sponsor_name":"LMU Klinikum","has_results":false},{"nct_id":"NCT03059264","title":"Trial Readiness and Endpoint Assessment in Congenital Myotonic Dystrophy","phase":"","overall_status":"COMPLETED","enrollment_count":100,"lead_sponsor_name":"Virginia Commonwealth University","has_results":false},{"nct_id":"NCT05006924","title":"Symptoms and Outcome Measures for Upper- Limb Function in Myotonic Dystrophy Type 1","phase":"","overall_status":"COMPLETED","enrollment_count":89,"lead_sponsor_name":"Oslo University Hospital","has_results":false},{"nct_id":"NCT06075693","title":"Cerebrospinal Fluid Biomarkers of Myotonic Dystrophy","phase":"","overall_status":"RECRUITING","enrollment_count":88,"lead_sponsor_name":"Massachusetts General Hospital","has_results":false},{"nct_id":"NCT04907162","title":"Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders","phase":"","overall_status":"COMPLETED","enrollment_count":82,"lead_sponsor_name":"LMU Klinikum","has_results":false},{"nct_id":"NCT05004129","title":"Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy","phase":"PHASE2, PHASE3","overall_status":"RECRUITING","enrollment_count":76,"lead_sponsor_name":"AMO Pharma Limited","has_results":false},{"nct_id":"NCT02375087","title":"Sleep Breathing Disorders, a Main Trigger for Cardiac ARythmias in Type I Myotonic Dystrophy ?","phase":"","overall_status":"COMPLETED","enrollment_count":73,"lead_sponsor_name":"University Hospital, Grenoble","has_results":false},{"nct_id":"NCT03784586","title":"Sudden Cardiac Death Stratification in Myotonic Dystrophy Type 1 Patients","phase":"","overall_status":"UNKNOWN","enrollment_count":70,"lead_sponsor_name":"University of Campania Luigi Vanvitelli","has_results":false},{"nct_id":"NCT03603171","title":"Clinical Outcome Measures in Myotonic Dystrophy Type 2","phase":"","overall_status":"COMPLETED","enrollment_count":60,"lead_sponsor_name":"Prof. Dr. Benedikt Schoser","has_results":false},{"nct_id":"NCT05848830","title":"Home-based Training and Supplementation in DM1 Patients","phase":"PHASE3","overall_status":"UNKNOWN","enrollment_count":60,"lead_sponsor_name":"McMaster University","has_results":false},{"nct_id":"NCT03692312","title":"Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy","phase":"PHASE2, PHASE3","overall_status":"COMPLETED","enrollment_count":56,"lead_sponsor_name":"AMO Pharma Limited","has_results":true},{"nct_id":"NCT04616807","title":"An Observational Study in Adult Patients With Non-dystrophic Myotonic Disorders","phase":"","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":53,"lead_sponsor_name":"Lupin Ltd.","has_results":false},{"nct_id":"NCT07362316","title":"Longitudinal Evaluation of Neuromuscular Involvement in Type 1 Myotonic Dystrophy","phase":"","overall_status":"COMPLETED","enrollment_count":50,"lead_sponsor_name":"Institut de Myologie, France","has_results":false},{"nct_id":"NCT03589677","title":"Brain Involvement in Myotonic Dystrophy Type I: From Functional Neuroimaging to the Impact on Quality of Life","phase":"","overall_status":"COMPLETED","enrollment_count":50,"lead_sponsor_name":"IRCCS San Camillo, Venezia, Italy","has_results":false},{"nct_id":"NCT04052958","title":"DM-IMT - Controlled, Randomized, Three-arm Intervention Study on the Safety and Efficacy of Regular Respiratory Muscle Training in Patients With Myotonic Dystrophy Type 1","phase":"NA","overall_status":"COMPLETED","enrollment_count":45,"lead_sponsor_name":"LMU Klinikum","has_results":false},{"nct_id":"NCT06926621","title":"A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I","phase":"PHASE2","overall_status":"ENROLLING_BY_INVITATION","enrollment_count":44,"lead_sponsor_name":"Vertex Pharmaceuticals Incorporated","has_results":false},{"nct_id":"NCT06185764","title":"A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)","phase":"PHASE1, PHASE2","overall_status":"RECRUITING","enrollment_count":44,"lead_sponsor_name":"Vertex Pharmaceuticals Incorporated","has_results":false},{"nct_id":"NCT04712422","title":"Poor Neck Proprioception May Cause Balance Deficits in Myotonic Dystrophy 1","phase":"","overall_status":"COMPLETED","enrollment_count":42,"lead_sponsor_name":"Istituto Auxologico Italiano","has_results":false},{"nct_id":"NCT07493525","title":"Fall Frequency and Factors Affecting Dynamic Balance in Patients With Myotonic Dystrophy Type 1","phase":"","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":41,"lead_sponsor_name":"Antalya Training and Research Hospital","has_results":false},{"nct_id":"NCT05072288","title":"A Remote Physical Activity Program in the Population Suffering from Type 1 Myotonic Dystrophy","phase":"NA","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":40,"lead_sponsor_name":"Université du Québec à Chicoutimi","has_results":false},{"nct_id":"NCT05027269","title":"Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients","phase":"PHASE1, PHASE2","overall_status":"COMPLETED","enrollment_count":39,"lead_sponsor_name":"Avidity Biosciences, Inc.","has_results":false},{"nct_id":"NCT05479981","title":"Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":37,"lead_sponsor_name":"Avidity Biosciences, Inc.","has_results":false},{"nct_id":"NCT04698551","title":"NIPD on cffDNA for Triplet Repeat Diseases","phase":"","overall_status":"UNKNOWN","enrollment_count":36,"lead_sponsor_name":"University Hospital, Montpellier","has_results":false},{"nct_id":"NCT04835298","title":"Breathlessness Assessment in Adult Patients With Myotonic Dystrophy Type 1","phase":"","overall_status":"COMPLETED","enrollment_count":34,"lead_sponsor_name":"CHU de Reims","has_results":false},{"nct_id":"NCT06844214","title":"A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 50 Years of Age With Non-congenital Myotonic Dystrophy Type 1","phase":"PHASE1, PHASE2","overall_status":"RECRUITING","enrollment_count":32,"lead_sponsor_name":"Sanofi","has_results":false},{"nct_id":"NCT04634682","title":"Effect of MYODM on Quality of Life, Fatigue and Hypersomnia in Patients With Myotonic Dystrophy Type 1","phase":"NA","overall_status":"COMPLETED","enrollment_count":30,"lead_sponsor_name":"Myogem Health Company, S.L.","has_results":false},{"nct_id":"NCT06378216","title":"Myotonic Dystrophy Type 1 Congenital and Juvenile Form: From Diagnosis to Rehabilitation [MDCJ-NeuBeRe]","phase":"","overall_status":"RECRUITING","enrollment_count":30,"lead_sponsor_name":"IRCCS Eugenio Medea","has_results":false},{"nct_id":"NCT03211923","title":"Muscle Relaxation in Myopathies With Positive Muscle Phenomena","phase":"","overall_status":"UNKNOWN","enrollment_count":30,"lead_sponsor_name":"Radboud University Medical Center","has_results":false},{"nct_id":"NCT07321977","title":"Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers","phase":"NA","overall_status":"RECRUITING","enrollment_count":30,"lead_sponsor_name":"Institut de Myologie, France","has_results":false},{"nct_id":"NCT07072676","title":"The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.","phase":"NA","overall_status":"ENROLLING_BY_INVITATION","enrollment_count":30,"lead_sponsor_name":"LMU Klinikum","has_results":false},{"nct_id":"NCT04886518","title":"Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":30,"lead_sponsor_name":"Harmony Biosciences Management, Inc.","has_results":true},{"nct_id":"NCT05890833","title":"The Risk of Falls Index for Patients With Neuromuscular Disorders","phase":"","overall_status":"COMPLETED","enrollment_count":30,"lead_sponsor_name":"LMU Klinikum","has_results":false},{"nct_id":"NCT06716931","title":"Investigating Exercise in Myotonic Dystrophy Type 2 (DM2)","phase":"NA","overall_status":"RECRUITING","enrollment_count":24,"lead_sponsor_name":"Massachusetts General Hospital","has_results":false},{"nct_id":"NCT06204809","title":"Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1","phase":"PHASE1","overall_status":"COMPLETED","enrollment_count":24,"lead_sponsor_name":"PepGen Inc","has_results":false},{"nct_id":"NCT05036447","title":"Myotonic Dystrophy Type 1 and Resistance Exercise","phase":"NA","overall_status":"COMPLETED","enrollment_count":21,"lead_sponsor_name":"Norwegian School of Sport Sciences","has_results":false},{"nct_id":"NCT06596850","title":"Wheelchair Skills Training for People with ARSACS and DM1","phase":"NA","overall_status":"NOT_YET_RECRUITING","enrollment_count":20,"lead_sponsor_name":"Laval University","has_results":false},{"nct_id":"NCT07075965","title":"Calcium Channel Blocker in Myotonic Dystrophy Type 1","phase":"PHASE1","overall_status":"NOT_YET_RECRUITING","enrollment_count":20,"lead_sponsor_name":"University of Rochester","has_results":false},{"nct_id":"NCT04187482","title":"Myotonic Dystrophy Type 1 Aerobic Exercise Study","phase":"NA","overall_status":"COMPLETED","enrollment_count":13,"lead_sponsor_name":"McMaster University","has_results":false},{"nct_id":"NCT04624750","title":"Open Label Study in Adolescents and Children With Myotonic Disorders","phase":"PHASE3","overall_status":"COMPLETED","enrollment_count":12,"lead_sponsor_name":"Lupin Ltd.","has_results":false}],"total":50},"guidelines":[],"source":"Drug Landscape verified database"}