Study of givinostat in young boys with Duchenne muscular dystrophy
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This trial tests an experimental drug called givinostat in boys ages 2 to under 6 years with Duchenne muscular dystrophy (DMD). The study looks at how the drug works in the body and if it is safe, with the goal of finding new treatment options for young children.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your son must be a boy between 2 and just under 6 years old.
- He must have a confirmed genetic diagnosis of Duchenne muscular dystrophy (DMD).
- If he takes steroids (like prednisone or deflazacort), the dose should have been stable for at least 3 months.
- He cannot have had certain other treatments, like gene therapy or experimental drugs, within the last few months.
- He must weigh at least 22 pounds (10 kilograms) at the start.
- He should be generally healthy aside from DMD, with no serious liver, kidney, or blood problems.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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