Gene editing trial for Duchenne muscular dystrophy (ages 2-7)
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This trial tests a new gene editing treatment called PBGENE-DMD for boys with Duchenne muscular dystrophy (DMD) whose genetic mutation falls within a specific range of the dystrophin gene (exons 45-55). The goal is to see if it is safe and can help improve muscle function.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be a boy between 2 and 7 years old.
- Your DMD diagnosis must be confirmed by genetic testing, with the mutation in exons 45 to 55 of the dystrophin gene.
- You must be able to walk at least 10 meters without help (if aged 2 to under 4) or at least 100 meters without help and have a certain motor function score (if aged 4 to 7).
- You cannot have had prior gene therapy, gene editing, or cell-based treatment.
- You must not have taken any experimental medication or drugs to increase dystrophin (like exon-skipping therapies) in the past 6 months.
- You must be free of antibodies to AAV9 (a virus used to deliver the treatment) and not have any condition that would prevent taking immune-suppressing medications.
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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