Efficacy & Safety of Tideglusib in Congenital Myotonic Dystrophy
Research type
Research Study
Full 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
IRAS ID
237950
Contact name
Stuart Evans
Contact email
Sponsor organisation
AMO Pharma Ltd.
Eudract number
2016-004623-23
Clinicaltrials.gov Identifier
130,741, IND No
Duration of Study in the UK
1 years, 0 months, 30 days
Research summary
Congenital Myotonic Dystrophy (CDM1) is a rare, life-threatening genetic disorder. Patients typically have a range of physical disabilities (including oral facial weakness, eating, respiratory and walking difficulties), are intellectually disabled and may have a co-occurring autism spectrum disorder. There are no approved treatments for CDM1.
There is evidence to suggest tideglusib may have potential to improve symptoms of CDM1, and this is a study of tideglusib versus placebo (dummy drug) across about a 5-month treatment period, in children and adolescents between the ages of 6 to 16 years with CDM1.
The purpose of the study is to investigate the efficacy and safety of tideglusib in this clinical population and to investigate the way tideglusib is absorbed and excreted from the body.
Prior to treatment there will be up to a 4 week screening period to ensure the subject is eligible. There will be a follow-up visit two weeks after the end of treatment for subjects who have not entered an extension study.
In total 56 subjects are planned to be enrolled in the UK, Canada and the United States.REC name
East Midlands - Leicester South Research Ethics Committee
REC reference
18/EM/0277
Date of REC Opinion
19 Nov 2018
REC opinion
Further Information Favourable Opinion