Gene therapy trial for children with OTC ammonia build-up
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This early-phase study tests an AAV gene therapy meant to reduce ammonia levels in children with ornithine transcarbamylase (OTC) deficiency. It’s designed to slow or stop ammonia-related harm, while also carefully monitoring safety for years.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You (or your child) must be 0–16 years old for study enrollment, with 6–16 for dose-adding parts
- OTC deficiency must be confirmed by a blood/tissue test or genetic testing
- The condition must be severe, meaning a restricted protein plan and at least one ammonia-lowering medicine
- Ammonia level before gene therapy must be below 100 µmol/L and not abnormal compared with past stable readings
- You must have a stable protein allowance and a stable ammonia-lowering medicine dose for the last 4 weeks
- You must agree to long-term safety follow-up for 4 more years
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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