drugset
drugset / Trial / NCT05004129

Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy

NCT05004129 ↗

Phase 2/3 Recruiting 76 enrolled AMO Pharma Limited
NaSingle-groupOpen-labelTreatment

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.

Timeline

Start
2021-08-23
Primary completion
2026-12-31
Completion
2026-12-31

Drugs

EvaluationDrugModalityDoseRoute
Subject Tideglusib Small molecule 400 mg Oral
Subject Tideglusib Small molecule 600 mg Oral
Subject Tideglusib Small molecule 800 mg Oral
Subject Tideglusib Small molecule 1000 mg Oral

Indications

congenital myotonic dystrophy