Congenital Myotonic Dystrophy Clinical Trials

11 clinical trials for Congenital Myotonic Dystrophy across international registries, including 7 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension — RECRUITING
• DMCRN-02-001: Assessing Pediatric Endpoints in DM1 — RECRUITING
• Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry — RECRUITING
• A PHASE 1/PHASE 2 OPEN-LABEL SINGLE ARM STUDY WITH DOSE ESCALATION (PART A), AND DOSE EXPANSION (PART B) PARTS TO EVALUATE THE SAFETY, TOLERABILITY, AND EFFICACY OF SAR446268, AN ADENO-ASSOCIATED VIRAL VECTOR-MEDIATED GENE THERAPY IN PARTICIPANTS 10 TO 50 YEARS OLD WITH NON-CONGENITAL MYOTONIC DYSTROPHY TYPE 1 — RECRUITING · PHASE2
• Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy — RECRUITING · PHASE2
• Myotonic Dystrophy Family Registry — RECRUITING
• A 52-WEEK, OPEN-LABEL STUDY TO EVALUATE THE LONG-TERM SAFETY AND EFFICACY OF TIDEGLUSIB FOR THE TREATMENT OF CONGENITAL DM1 (REACH CDM X) — RECRUITING
• Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy — COMPLETED · PHASE2
• Tideglusib: Expanded Access Use in Congenital Myotonic Dystrophy — OTHER
• Trial Readiness and Endpoint Assessment in Congenital Myotonic Dystrophy — COMPLETED
• A RANDOMIZED, DOUBLE-BLIND STUDY TO EVALUATE THE EFFICACY AND SAFETY OF TIDEGLUSIB VERSUS PLACEBO FOR THE TREATMENT OF CHILDREN AND ADOLESCENTS WITH CONGENITAL MYOTONIC DYSTROPHY — COMPLETED

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