Gene therapy for children with SPG47
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This trial tests a one-time gene therapy (BFB-101) for children with a rare genetic condition called SPG47, which causes problems with movement and development. The therapy aims to replace the faulty gene to help improve symptoms.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be between 12 months and 5 years old at the time of treatment.
- A doctor must have confirmed the SPG47 diagnosis through a genetic test.
- You must be able to travel to the study center and stay within 100 miles for the first 30 days after treatment.
- Your medications and treatments must have been stable for at least 3 months before the study starts.
- You must have received all recommended childhood vaccines.
- Your parents or guardians must be able to attend study visits and provide information about your health.
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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