Gene therapy study for children with SPG50
Part of Brain & nervous system clinical trials.
This study tests a gene therapy called IT MELPIDA for children with SPG50, a genetic condition that affects movement and development. The treatment is given through a lumbar puncture (spinal tap) and aims to slow or stop the loss of motor skills.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must have a confirmed diagnosis of SPG50 (a genetic change in the AP4M1 gene).
- Children must be between 4 months and 6 years old at the start of the study.
- Your child must show signs of nervous system problems (like trouble moving, speaking, or learning).
- If your child is on medications for muscle tightness, seizures, sleep, or behavior, the doses must be stable for at least 3 months.
- Your family must be willing to travel to the study center and live within 100 miles of it for 30 days after treatment.
- Your child should not have lost a major motor skill (like sitting, crawling, standing, walking, or climbing steps) in the last year.
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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