Tideglusib for Congenital or Childhood Onset Myotonic Dystrophy
This study is testing the safety and effectiveness of a drug called Tideglusib for individuals with Congenital Myotonic Dystrophy (a type of muscular dystrophy present at birth) or Childhood Onset Myotonic Dystrophy. Tideglusib will be given in different doses based on weight, starting at 400 mg and potentially increasing to 600 mg, 800 mg, or 1000 mg. The study is open to people aged 6 to 45 years old. To join, you must have previously participated in the AMO-02-MD-2-003 study, or be new to treatment for Congenital or Childhood Onset Myotonic Dystrophy. Researchers will primarily look at any side effects (adverse events) and use a special scale (Clinician-Completed Congenital DM1 Rating Scale) to see how well the treatment works over 52 weeks.
- Study design
- This is an open-label study, meaning both you and the study team will know you are receiving Tideglusib. It plans to enroll 76 participants.
- What's involved
- You would receive Tideglusib daily for 52 weeks, with a possible extension. Your dose would start at 400 mg and may increase over time.
- Compensation
- Not stated in the trial record.
- Follow-up
- Safety will be assessed for 52 weeks, with an optional extended access period where assessments continue every 8 weeks up to Week 132.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy
At a glance
Conditions
Where it's being run
14 sites across 11 statesStudy leadership
- Harriet Gray-Stephens, BM BCh, MA (Oxon), MFPM · STUDY_DIRECTOR · AMO Pharma
Who to contact
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What this trial measures
- Safety (Adverse Events)52 Weeks
The incidence of AEs, including SAEs, and abnormal findings in objective assessments (e.g. laboratory values, ECGs and vital signs) from Screening to Enrolment (where applicable), from Enrolment to End of Treatment (52 Weeks), and End of Treatment to the End of Follow-up period.
- Safety (Adverse Events) - With Optional Expanded AccessWeek 60 and every 8 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
The incidence of AEs, including SAEs, and abnormal findings in objective assessments (e.g. laboratory values, ECGs and vital signs) from End of Treatment to End of Optional Extended Access, and End of Optional Extended Access to the End of Follow-up period.
- Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)52 Weeks
The Clinician-completed Congenital DM1 Rating Scale is an 11-item rating scale completed by the clinician to score the symptom severity of domains that are clinically relevant in Congenital DM1.