Gene Therapy for Crigler Najjar Syndrome Type I (AlphaCN)

This is a Phase 1/2, multinational, open-label, study to evaluate the safety and efficacy of an intravenous infusion of GT-UGT1A1-AAV8-02 in patients with Crigler-Najjar type 1 aged ≤10 years and requiring phototherapy. Patients will received a single administration of GT-UGT1A1-AAV8-02 and will be followed for safety and efficacy of approximately 60 months (5 years): * a follow-up of approximately 12 months (48 weeks) * a long term follow-up of approximately 48 months (4 years), in order to be in line with the latest EMEA Guideline on follow-up of patients administered with gene therapy medicinal products, released on 22 Oct.2009 by the Committee for medicinal products for human use.

NCT Number	NCT06641154
Lead Sponsor	Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare
Conditions	Crigler-Najjar Syndrome Type I
Enrollment	5 participants
Start Date	2024-11-06
Primary Completion	2027-11-01 (estimated)
Study Completion	2029-11-01 (estimated)
Updated on ClinicalTrials.gov	2025-01-13