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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This Phase 3 trial tests givinostat to see if it can improve safety and functional outcomes in boys with Duchenne muscular dystrophy who cannot walk. You might be a candidate if you meet age, genetic diagnosis, wheelchair-level mobility, and specific arm-strength/respiratory/heart criteria.
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This trial looks at how safely givinostat works over a longer time in people with Duchenne muscular dystrophy (DMD). It mainly checks long-term side effects and tolerability, especially in a specific subgroup of muscle fat levels.
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This trial tests a new drug (NS-089/NCNP-02) that aims to help boys with Duchenne muscular dystrophy produce a shorter but still working version of the dystrophin protein. It's for boys who are still walking and have a specific genetic type that can be fixed by skipping a part of the gene called exon 44.
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