clinical trial · NCT03692312
Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy
Congenital Myotonic DystrophyTideglusibPlacebo
brief summary
This is a randomized, multicenter, double-blind, placebo-controlled, Phase 2/3 study of patients (aged 6 to 16 years) diagnosed with Congenital Myotonic Dystrophy (Congenital DM1).
started
Mar 3, 2021
primary completion
Apr 4, 2023
completion
Apr 4, 2023
last updated
Oct 8, 2025
detailed description
This is a randomized, double-blind, placebo controlled study of weight adjusted dose 1000 mg/day tideglusib versus placebo in the treatment of children and adolescents 6-16 years of age with Congenital DM1.
official title
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 (REACH CDM)
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol