Glycerol phenylbutyrate for children with urea cycle disorders
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a medicine called glycerol phenylbutyrate in children with urea cycle disorders, which are genetic conditions that make it hard for the body to get rid of extra ammonia. The goal is to see if the medicine can help lower ammonia levels and manage the condition safely.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Children from birth to 18 years old can join.
- You must have one of these urea cycle disorder types: CPS1 deficiency, ornithine translocase deficiency, citrullinemia type I, argininosuccinic aciduria, argininemia, or HHH syndrome.
- You have not taken glycerol phenylbutyrate in the past 3 months.
- If you are old enough to have children, you must agree to use effective birth control during the study and for 1 month after the last dose.
- You must be willing to follow the study's diet and visit schedule.
- You cannot be allergic to any ingredients in the medicine.
- You cannot take certain other drugs (like valproate or corticosteroids) within 24 hours before starting the study.
- You cannot be pregnant or breastfeeding.
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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