Gene therapy trial for a childhood AP4M1 brain disorder
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This trial studies a one-time gene therapy meant to help children with SPG50 disease caused by changes in the AP4M1 gene. Researchers will give the treatment and closely monitor safety and early signs of benefit.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child is between 4 months and 10 years old
- Your child has a confirmed AP4M1 genetic diagnosis (SPG50), with known disease-causing changes
- Your child has symptoms that fit SPG50 and includes problems with the nervous system
- Your child can do walking or leg movement tests: stand over 5 seconds, or take 5 steps (with help or a walker), or have mild ankle tightness (Ashworth 2 or less)
- You can give written permission for your child to take part in the study
- Your child can follow the study procedures (including scans and visits) as required
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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