Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn

This study is testing a gene therapy for children with OTC deficiency — a rare genetic liver disease where the body cannot properly break down protein, causing dangerous build-up of ammonia in the blood. The therapy uses a modified virus (AAV) to deliver a working copy of the gene into the liver. Researchers hope this one-time treatment can reduce or eliminate the need for lifelong dietary restrictions and medication. You may be eligible if: - The patient is 16 years old or younger (age 6–16 for the initial phase; 0–16 for the expansion phase) - OTC deficiency has been confirmed by enzyme or genetic testing - The patient has severe disease requiring protein restriction and ammonia-lowering medications - Ammonia levels are currently below 100 µmol/L and stable - The patient has been on stable medication and diet for the past 4 weeks You may NOT be eligible if: - The patient has high levels of antibodies against the AAV-LK03 virus vector - Liver enzyme levels (ALT, AST, bilirubin) are significantly elevated - There is evidence of serious liver disease (cirrhosis, liver failure, liver cancer) - The patient has active Hepatitis B or C, or HIV infection - The patient has had a liver transplant - The patient is pregnant or breastfeeding Talk to your doctor to see if this trial is right for you.