Gene therapy trial for children with SPG47 hereditary spastic paraplegia
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 SPG47, a genetic condition that affects movement and brain function. The therapy aims to deliver a working copy of the AP4B1 gene to help slow or stop the disease's progression.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Children aged 1 to 5 years old at the time of treatment
- A confirmed genetic diagnosis of SPG47 from a certified lab
- Symptoms of neurological problems based on medical history and exam
- Stable doses of any current medications (like for spasms, seizures, or sleep) for at least 3 months before screening
- Up to date on all CDC-recommended childhood vaccines
- Two parents or guardians who can give consent and attend all study visits
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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