Gene therapy study for baby boys with severe OTC deficiency
Part of Brain & nervous system, Digestive system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a gene therapy called ECUR-506 for baby boys under 9 months old with a severe form of OTC deficiency. The goal is to see if it can help their bodies process ammonia better, reducing the need for a strict protein-restricted diet and medications.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your baby is a boy and at least 37 weeks when born
- Age at screening is between 24 hours and 7 months old
- Weighs at least 3.5 kg (about 7.7 lbs) and no more than 13.5 kg (about 29.8 lbs)
- Has a confirmed genetic diagnosis of OTC deficiency with a severe form that caused a high ammonia crisis in the first week of life
- Is currently on both a low-protein diet and ammonia-scavenging medications
- Has received all age-appropriate vaccines
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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