NCT02858908

Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy

A Single-Blind, Phase 2 Study To Evaluate The Safety And Efficacy Of Tideglusib 400mg Or 1000mg For The Treatment Of Adolescent And Adult Congenital And Juvenile-Onset Myotonic Dystrophy

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 16 (actual)
• Sponsor: AMO Pharma Limited · Industry
• Sex: All
• Age: 12 Years – 45 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to determine whether Tideglusib is safe and efficacious in the treatment of adolescents and adults with congenital and juvenile-onset Myotonic Dystrophy. The pharmacokinetics of tideglusib and its primary metabolite will also be investigated.

Conditions

• Myotonic Dystrophy 1

Interventions

Type	Name	Description
DRUG	Tideglusib	Tideglusib for oral suspension,

Timeline

• Start date: 2016-07-20
• Primary completion: 2018-01-01
• Completion: 2018-01-01
• First posted: 2016-08-08
• Last updated: 2025-09-11
• Results posted: 2025-09-11

Locations

1 site across 1 country: United Kingdom

Regulatory

• FDA-regulated drug study