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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
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.
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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
Enrollment Count
17
Results Posted
Not Available
Therapy Information
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
Dose 2
5E12 vg/kg
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Na
Submit Date
2018-02-01
Completion Date
2030-03-30
Last Update
2023-03-28
Participation Criteria
Eligible Age
>=9 Years
Standard Ages
Child, Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
4
Locations
Netherlands,Italy,France
Regulatory Information
Has US IND
False
FDA Designations
Recent Updates
Resources/Links
Clinical Publications
Gene Therapy in Patients with the Crigler-Najjar Syndrome
Preclinical Publications
Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome
A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome
Protocol
Clinical Trial Protocol