Ignite Creation Date:
2024-05-06 @ 11:12 AM
Last Modification Date:
2024-10-26 @ 12:42 PM
Study NCT ID:
NCT03466463
Status:
RECRUITING
Last Update Posted:
2023-03-28
First Post:
2018-02-01
Brief Title:
Gene Therapy for Severe Crigler Najjar Syndrome
Sponsor:
Genethon
Organization:
Genethon
Study Overview
Official Title:
A Phase III 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
Status Verified Date:
2023-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
CareCN
Brief Summary:
This is a Phase 12 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 Oct2009 by the Committee for medicinal products for human use
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 Oct2009 by the Committee for medicinal products for human use
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None