Gene Therapy for Severe Crigler Najjar Syndrome

NCTID	NCT03466463 (View at clinicaltrials.gov)
Description	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. (Show More)
Development Status	Active
Indication	Crigler-Najjar Syndrome
Disease Ontology Term	DOID:3803
Compound Name	GNT0003
Compound Description	ssAAV8.hUGT1A1
Sponsor	Genethon
Funder Type	Other
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	17 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	UGT1A1
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell	Liver
Delivery System	Viral transduction
Vector Type	AAV8
Editor Type	none
Dose 1	2E12 vg/kg (n=2)
Dose 2	5E12 vg/kg (n=3)

Eligible Age	>=9 Years
Standard Ages	Child, Adult, Older adult
Sexes Eligible for Study	ALL
Eligibility Criteria	Inclusion Criteria: * 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 Criteria: * 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 View Inclusion and Exclusion Criteria at ClinicalTrials.gov

No.of Trial Sites	4
Locations	Netherlands,Italy,France

Has US IND	False
FDA Designations
Recent Updates	Pivotal part of the trial has been initiated