Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy
Trial Parameters
Brief Summary
This is an open-label phase 2/3 study for individuals with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who are treatment naïve.
Eligibility Criteria
Inclusion Criteria: Subjects who do not enter this study directly from completing the AMO-02-MD-2-003 study (i.e. subjects who did not complete AMO-02-MD-2-003, subjects who completed AMO-02-MD-2-003 but did not directly rollover or subjects who are re-entering AMO-02-MD-2-004), will not be considered eligible for the study without meeting all of the criteria below: 1. Subjects under study must be individuals with a diagnosis of Congenital or Childhood Onset DM1. 2. Diagnosis must be genetically confirmed 3. Subjects must be male or female aged ≥6 years to ≤45 years at Screening 4. Subjects must have a Clinical Global Impression - Severity (CGI-S) score of 3 or greater at Screening (V-1) 5. Written, voluntary informed consent must be obtained before any study related procedures are conducted. Where a parent or legally authorized representative (LAR) provides consent, there must also be assent from the subject (as required by local regulations) 6. Subject's caregiver must be willing and