Contact SCGE




Clinical Trial Report

Gene Therapy Trial Report

Summary

Gene Therapy for Severe Crigler Najjar Syndrome


NCTID NCT03466463 (View at clinicaltrials.gov)
Description
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