Testing LE051 for boys with Duchenne muscular dystrophy (exon 51)
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This study tests a new medicine called LE051 for boys with Duchenne muscular dystrophy (DMD) who have a specific genetic change that allows 'exon 51 skipping.' The goal is to see if it is safe and can help improve muscle function. Boys who join will be closely monitored for heart and lung health.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are a boy between 4 and 8 years old.
- Your genetic test shows you have a type of DMD that can be treated by skipping a part of the gene called exon 51.
- You and your parent or guardian agree to take part in the study and sign the consent form.
- Your heart is pumping well (LVEF is 40% or higher).
- Your lungs are working well enough (FVC is 40% or more of the predicted value).
- Your heart's electrical activity (QTc) is within a safe range.
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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