Found 76 matching trials
Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1 PHASE2 The primary objective of this study is to evaluate the safety and efficacy ...
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UCI Center for Clinical Research
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PhenoDM1 (Myotonic Dystrophy Type 1 Natural History Study) N/A PhenoDM1 will use patient reported outcomes to assess levels of pain, fatig...
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Newcastle-upon-Tyne Hospitals NHS Trust
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Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy PHASE1 This is a phase 1/2a double-blinded, placebo-controlled, dose-escalating st...
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Liverpool Hospital
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The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2 PHASE3 A Randomized, Double-blind, Placebo-Controlled, Multi-Center Study to Inves...
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Laboratory for Muscle Diseases and Neuropathies
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Evaluation of Cognitive Functions in 20 Patients With Type 1 Myotonic Dystrophy With Virtual Reality Approach NA Type 1 myotonic dystrophy (MD1) is a genetic and hereditary disease that pr...
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Multiple Locations
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Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients PHASE1 AOC 1001-CS1 is a randomized, double-blind, placebo-controlled, Phase 1/2 s...
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University of California Los Angeles
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A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I PHASE2 The purpose of the study is to evaluate the long-term safety and tolerabili...
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Enrolling By Invitation
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St. Louis Children's Hospital - Neurology
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Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1 PHASE3 The purpose of the study is to assess the efficacy, safety, and tolerabilit...
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UCSD - Altman Clinical and Translational Research Institute
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Efficacy and Tolerance of AVAPS Mode in Myotonic Dystrophy NA Myotonic dystrophy type 1 is a myopathy with complex respiratory pattern an...
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Home ventilation Unit , Raymond Poincaré hospital
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Safety and Efficacy Study of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) In Myotonic Dystrophy Type 1 PHASE2 To investigate the effects of rhIGF-I/rhIGFBP-3 treatment for 24 weeks on e...
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University of California Irvine Medical Center; MDA, ALS and Neuromuscular Center
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