NCT05004129

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)

• Status: Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 76 (estimated)
• Sponsor: AMO Pharma Limited · Industry
• Sex: All
• Age: 6 Years – 45 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This is an open-label study of either a weight-adjusted 1000 mg fixed dose or a weight banded fixed dose of tideglusib across a 52-week treatment period with an open-ended optional extended access period. The subjects are children and adolescents with Congenital DM1 who participated in the antecedent AMO-02-MD-2-003 study or individuals with either Congenital or Childhood onset DM1 who are treatment naïve.

Conditions

• Congenital Myotonic Dystrophy

Interventions

Type	Name	Description
DRUG	Tideglusib	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.

Timeline

• Start date: 2021-08-23
• Primary completion: 2026-12-31
• Completion: 2026-12-31
• First posted: 2021-08-13
• Last updated: 2025-05-28

Locations

14 sites across 4 countries: United States, Australia, Canada, New Zealand

Regulatory

• FDA-regulated drug study