Efficacy and Safety of Givinostat in Duchenne Muscular Dystrophy
Research type
Research Study
Full title
Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.
IRAS ID
210215
Contact name
Michela Guglieri
Contact email
Sponsor organisation
Italfarmaco S.p.A.
Eudract number
2016-000401-36
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
126598, IND Number:
Duration of Study in the UK
3 years, 2 months, 31 days
Research summary
This is a Randomised, Double-blind, Placebo-controlled study to evaluate the efficacy and safety of givinostat (investigational product) in patients with Duchenne Muscular Dystrophy (DMD) who can walk. About 192 participants, boys (aged 6 years or older) and adult men, diagnosed with DMD, are anticipated to take part in this study. the study is being conducted in some countries in Europe, and in North America.
Participants will take part in this study for about 20 months. Participants will be expected to make 16 visits to their study centre.
The study is divided into 2 time periods: a screening period (which will start about 4 weeks before the participant receives their first dose of study drug) and a treatment period (which will include 18 months of treatment with the study drug). A follow up visit will take place 4 weeks after the final Treatment visit, if the participant decides not to take part, or is not eligible, for the long term study.
There will be two treatment groups for this study, one group will receive givinostat, and the other group will receive placebo. Participants will be randomly assigned to one of these two groups on a 2:1 basis (givinostat:placebo).
The study drug (givinostat or placebo) will be provided in a liquid, to be taken by mouth, twice a day, after a meal. The dose of the study drug will be calculated based on the weight of the participant.
REC name
North East - Newcastle & North Tyneside 2 Research Ethics Committee
REC reference
16/NE/0413
Date of REC Opinion
7 Mar 2017
REC opinion
Further Information Favourable Opinion