Safety & Efficacy of Tideglusib in Type 1 Myotonic Dystrophy
Research type
Research Study
Full title
A Single-Blind, Phase 2 Study To Evaluate The Safety And Efficacy Of Tideglusib 400 mg Or 1000 mg For The Treatment Of Adolescent And Adult Congenital And Juvenile-Onset Myotonic Dystrophy
IRAS ID
199733
Contact name
Gráinne Gorman
Contact email
Sponsor organisation
AMO Pharma Ltd.
Eudract number
2016-000067-16
Duration of Study in the UK
1 years, 1 months, 0 days
Research summary
This is a 20 week study in adolescent and adult subjects with a genetic diagnosis of type 1 myotonic dystrophy (DM-1) since birth or since before the age of 12 years old.
The purpose of the study is firstly to investigate the safety and tolerability of tideglusib in this clinical population. Secondly, the study is to investigate changes in outcomes that reflect if tideglusib is effective for treating the symptoms of DM-1, since birth or since before the age of 12, and to investigate the way tideglusib is absorbed and excreted from the body.
Two dose levels will be investigated: 1000mg for the first 8 subjects and then following review by a safety committee, 400mg for the next 8 subjects. In total, 16 subjects are planned to be enrolled.
The study will involve 14 weeks of treatment with either study drug tideglusib or placebo for each subject. All subjects will receive both active and placebo but, the subject and their caregiver will be unaware when they are receiving study drug and placebo during this 14 week period (single-blind). Prior to treatment, there will be up to a 4 week screening period to determine if the subject is eligible. After treatment, there will be a 2 week follow-up period. The study will involve a number of cognitive and muscle function tests as well as lung function tests and patient/caregiver reported outcome questionnaires.REC name
North East - Newcastle & North Tyneside 2 Research Ethics Committee
REC reference
16/NE/0057
Date of REC Opinion
20 Apr 2016
REC opinion
Further Information Favourable Opinion