NCT03692312

Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

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)

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 56 (actual)
• Sponsor: AMO Pharma Limited · Industry
• Sex: All
• Age: 6 Years – 16 Years
• Healthy volunteers: Not accepted

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).

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.

Conditions

• Congenital Myotonic Dystrophy

Interventions

Type	Name	Description
DRUG	Tideglusib	Tideglusib for oral suspension, weight-adjusted at 400mg, 600mg or 1000 mg dose levels, once daily
DRUG	Placebo	Matching placebo formulation

Timeline

• Start date: 2021-03-03
• Primary completion: 2023-04-04
• Completion: 2023-04-04
• First posted: 2018-10-02
• Last updated: 2025-10-08
• Results posted: 2025-09-11

Locations

14 sites across 5 countries: United States, Australia, Canada, New Zealand, United Kingdom

Regulatory

• FDA-regulated drug study