Gene Therapy for Crigler Najjar Syndrome Type I (AlphaCN)
Status:
RECRUITING
Trial end date:
2029-11-01
Target enrollment:
Participant gender:
Summary
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.
Phase:
PHASE1
Details
Lead Sponsor:
Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare