Randomised, double-blind, placebo-controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy EPIDYS (Epigenetic Rescue of Dystrophin Dysfunction)
The main purpose of this study is to learn how well the investigational drug givinostat works and how safe givinostat is compared to placebo in males 6 years old to 17 years old.
A placebo is an inactive material that looks like the study drug but does not contain any active study drug. Researchers use a placebo to see if the study drug works better or is safer than not taking anything. There is a 1 in 3 chance (33%) that your child may receive placebo. Both givinostat and placebo will be referred to as “study drug”.
The study is divided into 2 time periods: a screening period (which will start about 4 weeks before your child receives the first dose of study drug) and a treatment period (which will include 18 months of treatment with the study drug). At the end of the 18-month treatment period of this study, you will be asked to have your child participate in a long-term study.
- 6-17 years old
- Diagnosed with Duchenne Muscular Dystrophy
Additional inclusion and exclusion criteria apply. Please contact Cristina Liberati for more information:
- (352) 294-8706
Can be done from home
KeywordsMuscular dystrophy, Muscular dystrophy - resources, Neurology
Principal InvestigatorBarry Byrne, MD, PhD
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Studies listed on this site have been approved by a UF Institutional Review Board (IRB), which works to ensure the welfare and rights of research participants as required by federal regulations. Study listings are provided by the UF Clinical and Translational Science Institute in collaboration with UF research teams and the UF IRBs.