Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy

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.

Timeline

Start

2016-07-20

Primary completion

2018-01

Completion

2018-01

Publications

• Horrigan J, Gomes TB, Snape M, Nikolenko N, McMorn A, Evans S, Yaroshinsky A, Della Pasqua O, Oosterholt S, Lochmuller H. A Phase 2 Study of AMO-02 (Tideglusib) in Congenital and Childhood-Onset Myotonic Dystrophy Type 1 (DM1). Pediatr Neurol. 2020 Nov;112:84-93. doi: 10.1016/j.pediatrneurol.2020.08.001. Epub 2020 Aug 5.

Drugs

Indications