Gene therapy safety study for non-ambulatory Duchenne
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This Phase 1 trial studies a one-time gene therapy treatment (SRP-9001) to see if it is safe and whether it can produce the needed gene activity in people with Duchenne muscular dystrophy (DMD) who are not walking. It may help researchers understand whether this approach could be useful for DMD patients, especially in later stages.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a confirmed diagnosis of Duchenne muscular dystrophy (DMD) from prior testing and clinical findings
- You are in the non-walking group for this study and meet the study’s specific non-ambulatory criteria at screening
- Your upper-limb function scores at screening must fall in the required range (PUL entry score at least 3; total PUL score 20 to 40)
- You must be able to cooperate with physical/motor testing during visits
- Your blood test for certain antibodies (rAAVrh74 antibody titers) must not be too high
- You must not have certain health issues (like autoimmune disease or other treatments/conditions) that could raise the risk from gene therapy
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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