Study of GT-UGT1A1-AAV8-02 Gene Therapy by Intravenous Infusion in Children with Crigler-Najjar Syndrome Type I Requiring Phototherapy

What this Trial Is About

Researchers are evaluating the safety and effectiveness of a gene therapy called GT-UGT1A1-AAV8-02 for patients with Crigler-Najjar syndrome type I, a rare inherited condition causing severe jaundice and neurologic problems due to a genetic enzyme deficiency. This condition typically requires lifelong phototherapy to manage bilirubin levels, and currently, liver transplantation is the only specific treatment. The study focuses on patients aged 3 months to 10 years who need phototherapy, aiming to reduce bilirubin levels and improve health without the need for ongoing phototherapy. Participants will receive a single intravenous infusion of GT-UGT1A1-AAV8-02, which uses a genetically engineered virus to deliver a healthy copy of the UGT1A1 gene to liver cells. This gene therapy is designed to restore the enzyme's function to process bilirubin properly. The study includes an initial follow-up period of about 12 months (48 weeks) to monitor safety and treatment effects, followed by a long-term follow-up of approximately 4 years to ensure ongoing safety and efficacy in line with regulatory guidelines. During the study, patients will be closely monitored through laboratory tests that measure bilirubin levels and liver function, as well as assessments of any side effects or adverse events. The main outcomes include the proportion of patients achieving target bilirubin levels without phototherapy and the occurrence of any treatment-related adverse events over 48 weeks. The total participation duration is about 5 years, allowing comprehensive evaluation of the gene therapy's impact and safety over time.

Gene Therapy for Crigler Najjar Syndrome Type I (AlphaCN)