Phase 12 Study of Intravenous GT-UGT1A1-AAV8-02 Gene Therapy for Children With Crigler-Najjar Syndrome Type I Who Need Phototherapy

What this Trial Is About

This research aims to evaluate the safety and effects of an intravenous infusion of GT-UGT1A1-AAV8-02 in patients with Crigler-Najjar syndrome type I, a rare inherited liver enzyme disorder causing severe jaundice and neurological problems. This condition affects children up to 10 years old who require phototherapy. Because there is no specific treatment besides liver transplantation, this study tests gene therapy as a possible alternative. The study is a Phase 12, open-label, multinational trial. Participants will receive a single dose of GT-UGT1A1-AAV8-02, a gene therapy using a modified virus to deliver a functioning UGT1A1 gene to the liver. The study includes two dose levels assessed in a dose escalation design. After treatment, participants will be followed for safety and effectiveness over about 5 years 12 months for initial follow-up and an additional 4 years for long-term monitoring according to regulatory guidelines. During the study, participants will undergo regular assessments of bilirubin levels to measure treatment impact, evaluate health-related quality of life, and monitor for any adverse effects. Safety will be closely observed through laboratory tests and clinical evaluations. The main outcomes include the proportion of patients achieving bilirubin control without phototherapy within 48 weeks and tracking treatment-related side effects. The total study duration for each participant will be approximately 5 years to ensure thorough safety and efficacy data collection.

Gene Therapy for Crigler Najjar Syndrome Type I (AlphaCN)