Myotonic Dystrophy Clinical Trials

45 clinical trials for Myotonic Dystrophy across international registries, including 25 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1 — RECRUITING · PHASE3
• Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension — RECRUITING
• Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension — RECRUITING
• Global Open-Label Extension Study of Del-desiran for the Treatment of DM1 — RECRUITING · PHASE3
• 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 — RECRUITING · PHASE1
• Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers — RECRUITING · NA
• Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders — RECRUITING
• The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2 — RECRUITING · PHASE3
• 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. — RECRUITING · PHASE3
• Development of Quantitative Muscle Imaging as a Biomarker of Disease Endpoints in Myotonic Dystrophy — RECRUITING
• A PHASE 1, OPEN-LABEL TRIAL TO ASSESS THE SAFETY AND PHARMACOKINETICS OF MULTIPLE ASCENDING DOSES OF DT-818 IN HEALTHY VOLUNTEERS AND PARTICIPANTS WITH MYOTONIC DYSTROPHY TYPE 1 — RECRUITING · PHASE1
• Biomarker Development for Muscular Dystrophies — RECRUITING
• Extracellular RNA Biomarkers of Myotonic Dystrophy — RECRUITING
• Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry — RECRUITING
• Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders — RECRUITING
• A GLOBAL PHASE 3 OPEN-LABEL EXTENSION STUDY TO ASSESS THE LONG-TERM SAFETY, TOLERABILITY, AND EFFICACY OF INTRAVENOUS AOC 1001 FOR THE TREATMENT OF MYOTONIC DYSTROPHY TYPE 1 — RECRUITING · PHASE3
• AN OPEN-LABEL EXTENSION STUDY EVALUATING SAFETY AND PHARMACOKINETICS IN PARTICIPANTS WITH MYOTONIC DYSTROPHY TYPE 1 (FREEDOM-OLE) — RECRUITING
• Myotonic Dystrophy Family Registry — RECRUITING
• A PHASE 3 RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, GLOBAL STUDY TO EVALUATE THE EFFICACY AND SAFETY OF INTRAVENOUS AOC 1001 FOR THE TREATMENT OF MYOTONIC DYSTROPHY TYPE 1 — RECRUITING · PHASE3
• A PHASE 2 RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE-ASCENDING DOSE STUDY OF PGN-EDODM1 IN ADULT PARTICIPANTS WITH MYOTONIC DYSTROPHY TYPE 1 (FREEDOM2-DM1) — RECRUITING · PHASE2
• A PHASE 1/2A DOSE-ESCALATING STUDY TO EVALUATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF ARO-DM1 (SRP-1003) IN SUBJECTS WITH TYPE 1 MYOTONIC DYSTROPHY WHO ARE 18 TO 65 YEARS — RECRUITING · PHASE1
• A PHASE 1/2A DOSE-ESCALATING STUDY TO EVALUATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF ARO-DM1 IN SUBJECTS WITH TYPE 1 MYOTONIC DYSTROPHY WHO ARE 18 TO 65 YEARS — RECRUITING · PHASE1
• A PHASE 1/2A DOUBLE-BLIND, PLACEBO-CONTROLLED, SINGLE- AND MULTIPLE ASCENDING DOSE STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND PRELIMINARY CLINICAL EFFICACY OF INTRAVENOUS ADMINISTRATION OF ATX-01 IN MALE AND FEMALE PARTICIPANTS AGED 18 TO 64 WITH CLASSIC MYOTONIC DYSTROPHY TYPE 1 (DM1) — RECRUITING · PHASE1
• The United Kingdom National Registry for Myotonic Dystrophy — RECRUITING
• A PHASE 1/2, RANDOMIZED, DOUBLE-BLIND, PLACEBO¿CONTROLLED SINGLE- AND MULTIPLE¿DOSE ESCALATION STUDY EVALUATING THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF VX¿670 IN ADULT SUBJECTS WITH MYOTONIC DYSTROPHY TYPE 1 — RECRUITING · PHASE1
• A Phase 1/2, Randomized, Double-blind, Placebo‑controlled Single- and Multiple‑dose Escalation Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VX‑670 in Adult Subjects with Myotonic Dystrophy Type 1 — TERMINATED · PHASE2
• An Observational Study in Adult Patients With Non-dystrophic Myotonic Disorders — COMPLETED
• An Open-label Extension Study Evaluating the Long-term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects with Myotonic Dystrophy Type I — TERMINATED · PHASE2
• RAMYD Study - Evaluation of Arrhythmic Risk in Myotonic Dystrophy — COMPLETED · NA
• Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients — COMPLETED · PHASE1
• Global Study of Del-desiran for the Treatment of DM1 — ACTIVE_NOT_RECRUITING · PHASE3
• Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients — COMPLETED · PHASE2
• Open Label Study in Adolescents and Children With Myotonic Disorders — COMPLETED · PHASE3
• The Risk of Falls Index for Patients With Neuromuscular Disorders — COMPLETED
• At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions — COMPLETED
• A PHASE 1 PLACEBO-CONTROLLED STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF SINGLE-ASCENDING DOSES OF PGN-EDODM1 IN ADULT PARTICIPANTS WITH MYOTONIC DYSTROPHY TYPE 1 — COMPLETED · PHASE1
• A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PITOLISANT ON EXCESSIVE DAYTIME SLEEPINESS AND OTHER NON-MUSCULAR SYMPTOMS IN PATIENTS WITH MYOTONIC DYSTROPHY TYPE 1, FOLLOWED BY AN OPEN-LABEL EXTENSION — COMPLETED
• Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1 — COMPLETED · PHASE1
• European Home Mechanical Ventilation Registry — TERMINATED
• Sleep Breathing Disorders, a Main Trigger for Cardiac ARythmias in Type I Myotonic Dystrophy ? — COMPLETED
• Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1 — COMPLETED · PHASE2
• Effects of SomatoKine (Iplex)Recombinant Human Insulin-like Growth Factor-1/Recombinant Human Insulin-like Growth Factor-binding Protein-3 (rhIGF-I/rhIGFBP-3) in Myotonic Dystrophy Type 1 (DM1) — COMPLETED · PHASE1
• DM1 Heart Registry - DM1 Respiratory Registry — COMPLETED
• Postural Spirometry Changes in Ambulatory Myotonic Dystrophy Patients — COMPLETED · NA
• Efficacy and Safety of DHEA for Myotonic Dystrophy — COMPLETED · PHASE2

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