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
Genethon
17 participants
Mar 19, 2018
INTERVENTIONAL
Conditions
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.
Eligibility
Inclusion Criteria3
- Patients with severe Crigler-Najjar syndrome resulting from a molecular confirmation of mutations in the UGT1A1 gene and requiring phototherapy
- Male or female at least 9 years at the date of signature of informed consent
- Patient able to give informed assent and/or consent in writing
Exclusion Criteria7
- Patients who underwent liver transplantation
- Patients with chronic hepatitis B or C
- Patients infected with Human immunodeficiency virus (HIV)
- Patients with significant underlying liver disease
- Patients with significant encephalopathy
- Participation in any other investigational trial during this trial
- Patients unable or unwilling to comply with the protocol requirements
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Interventions
Intravenous infusion, single dose
Locations(4)
View Full Details on ClinicalTrials.gov
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NCT03466463