Long-Term Safety and Effectiveness Study of Tideglusib for Children and Adults with Congenital or Childhood Onset Myotonic Dystrophy

What this Trial Is About

Researchers are conducting an open-label phase 23 study to evaluate tideglusib in children and adolescents with Congenital or Childhood Onset Myotonic Dystrophy DM1. This includes those who participated in a previous related study or are treatment-nave. The study aims to assess long-term safety and efficacy in this specific group affected by Congenital DM1. Participants will receive tideglusib orally once daily, either as a weight-adjusted dose of 400 mg, 600 mg, or 1000 mg, or a weight banded fixed dose ranging from 400 mg to 1000 mg. All subjects start at 400 mg for two weeks, then increase to 600 mg for the next two weeks. The treatment period lasts 52 weeks, followed by an optional extended access phase that continues beyond the initial treatment period. During the study, participants will undergo various assessments including safety monitoring through adverse event tracking, and evaluations using the Clinician-Completed Congenital DM1 Rating Scale, Clinical Global Impressions scales, caregiver questionnaires, and physical tests like the 10-meter walk-run test. Blood tests will measure CTG repeats and plasma troponin T levels. These evaluations continue at regular intervals during the treatment and extended access phases, which may last up to 132 weeks or until study closure.

Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy