Gene therapy trial for Duchenne muscular dystrophy in young boys
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This trial tests a new gene therapy called SPOT-03 to see if it can help the body make dystrophin, a protein that is missing in Duchenne muscular dystrophy (DMD). It is for boys ages 2 to under 8 who can still walk at least 10 meters.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Boys aged 2 to under 8 years old
- A confirmed diagnosis of Duchenne muscular dystrophy from genetic testing
- Able to walk at least 10 meters without help
- Healthy enough to have a small muscle biopsy under anesthesia
- Good heart, lung, liver, and kidney function (e.g., ejection fraction of 50% or higher, lung capacity above 50% expected, no need for nighttime breathing support)
- No use of other gene therapy or investigational drugs in the past
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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