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)

Purpose

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”.

Procedures

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. 

Eligibility

  • Male
  • 6-17 years old 
  • Diagnosed with Duchenne Muscular Dystrophy

Additional inclusion and exclusion criteria apply. Please contact Cristina Liberati for more information: 

Age

18 and under

Gender

Male

Monetary compensation

No

Can be done from home

No

Keywords

Muscular dystrophy, Muscular dystrophy - resources, Neurology

Principal Investigator

Barry Byrne, MD, PhD

Department

Neurology

Contact Information

c.liberati@peds.ufl.edu

(352) 294-8706


Be an Informed Participant

Before deciding to participate in a research study, take time to learn about clinical research, how it's conducted and your rights as a research participant. Following are some helpful resources from independent sources. Always remember that a clinical research study is research, not treatment.

Know Who to Contact

  • Eligibility: For questions about a specific study and who is eligible to participate, call or email the contact person listed for that study.
  • Your Rights: For questions about your rights as a research participant, contact the UF Institutional Review Boards at 352-273-9600.
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