Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy — REACH CDM X  

Congenital Myotonic Dystrophy Clinical Trial

Official title: 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

Clinical Trial 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.

Clinical Trial Description

This is an open-label study of weight-adjusted 1000 mg tideglusib, once daily for 52 weeks with an open-ended optional extended access period in children and adolescents with a diagnosis of Congenital DM1 who participated in the AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who are treatment naïve. ;

Related Conditions & MeSH terms

• Congenital Myotonic Dystrophy
• Myotonic Dystrophy

• NCT number: NCT05004129
• Study type: Interventional
• Source: AMO Pharma Limited
• Status: Recruiting
• Phase: Phase 2/Phase 3
• Start date: August 23, 2021
• Completion date: March 28, 2025