Base editing study for severe urea cycle disorders
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a new medicine called LNP.UCD.ABE that aims to fix a specific genetic change causing a severe urea cycle disorder. It uses a technique called base editing to correct the gene mutation, which may help your body break down ammonia better.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a severe urea cycle disorder diagnosed by your doctor.
- Genetic testing must show a specific mutation in the CPS1 gene that the study drug is designed to fix.
- You need to have had a history of very high ammonia levels before age 1, unless diagnosed before birth and treated right away.
- If you were diagnosed more than 8 weeks ago, you must still need protein restriction and ammonia-lowering medicine, or have had a hyperammonemic event or seizure caused by high ammonia.
- You must weigh more than 3.5 kilograms (about 7.7 pounds) at the time of screening.
- You cannot have had a liver transplant, be in need of urgent liver transplant, or have participated in another gene therapy trial 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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