Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy
An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Tideglusib for the Treatment of Congenital or Childhood Onset DM1 (REACH CDM X)
AMO Pharma Limited
76 participants
Aug 23, 2021
INTERVENTIONAL
Conditions
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
Plain Language Summary
Simplified for easier understanding
This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.
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Interventions
Tideglusib dosing will be weight-adjusted at 400 mg, 600 mg, or 1000 mg dose levels, or weight banded fixed doses of 400 mg, 600 mg, 800 mg or 1000 mg, with each subject starting at a weight-adjusted 400 mg dose level for 2 weeks, then up titrating to a weight-adjusted 600 mg dose level for the next 2 weeks.
Locations(14)
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NCT05004129