Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

Congenital Myotonic Dystrophy Clinical Trial

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)

Status Completed

Clinical Trial 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).

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT03692312
Study type	Interventional
Source	AMO Pharma Limited
Contact
Status	Completed
Phase	Phase 2/Phase 3
Start date	March 3, 2021
Completion date	April 4, 2023

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See also
Status	Clinical Trial	Phase
Recruiting	`NCT00082108` - Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry
Recruiting	`NCT02398786` - Myotonic Dystrophy Family Registry
Recruiting	`NCT03059264` - Trial Readiness and Endpoint Assessment in Congenital Myotonic Dystrophy
Recruiting	`NCT05224778` - DMCRN-02-001: Assessing Pediatric Endpoints in DM1
Recruiting	`NCT05004129` - Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy	Phase 2/Phase 3