Dose-check study for children with AADC deficiency using brain surgery
Part of Brain & nervous system, Genetic & congenital clinical trials.
This Phase 1 trial tests how safe different treatment doses are for children with AADC deficiency, using MRI-guided brain surgery. It may help improve movement and developmental progress after standard medicines did not work well enough.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a definite AADC deficiency diagnosis confirmed by specific lab tests or genetic testing
- Your child is at least 24 months old
- Standard treatments have not helped enough (ongoing eye movement crises and developmental delays)
- Your child has a significant delay in walking (not able to walk independently by 18 months)
- Your child must be healthy enough for MRI-guided surgery and sedation (including normal enough blood/clotting tests)
- Parents must agree to frequent, long follow-up visits and provide consent
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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