Gene Therapy for Severe Crigler Najjar Syndrome — CareCN  

Crigler-Najjar Syndrome Clinical Trial

Official title: A Phase I/II, Open Label, Study to Evaluate Safety and Efficacy of an Intravenous Injection of GNT0003 (AAV Vector Expressing the UGT1A1 Transgene) in Patients With Severe Crigler-Najjar Syndrome Requiring Phototherapy

Status Recruiting

Clinical Trial Summary

This is a Phase 1/2, multinational, open-label, study to evaluate the safety and efficacy of an intravenous infusion of GNT0003 in patients with Crigler-Najjar aged ≥10 years and requiring phototherapy. Patients will received a single administration of GNT0003 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.

Clinical Trial Description

n/a

Related Conditions & MeSH terms

• Cardiomyopathies
• Crigler-Najjar Syndrome
• Syndrome

• NCT number: NCT03466463
• Study type: Interventional
• Source: Genethon
• Contact: Genethon Clinical Development Department
• Phone: 00 33 (0)1 69 47 10 32
• Email: clinical_development@genethon.fr
• Status: Recruiting
• Phase: N/A
• Start date: March 19, 2018
• Completion date: March 30, 2030