NCT03466463

Gene Therapy for Severe Crigler Najjar Syndrome

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
• Phase: N/A
• Study type: Interventional
• Enrollment: 17 (estimated)
• Sponsor: Genethon · Academic / Other
• Sex: All
• Age: 9 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Crigler-Najjar Syndrome

Interventions

Type	Name	Description
GENETIC	GNT0003	Intravenous infusion, single dose

Timeline

• Start date: 2018-03-19
• Primary completion: 2026-03-30
• Completion: 2030-03-30
• First posted: 2018-03-15
• Last updated: 2023-03-28

Locations

4 sites across 3 countries: France, Italy, Netherlands