Ignite Creation Date:
2025-12-25 @ 2:50 AM
Ignite Modification Date:
2026-01-22 @ 1:58 PM
Study NCT ID:
NCT06687733
Status:
RECRUITING
Last Update Posted:
2024-11-14
First Post:
2024-11-11
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Safety and Efficacy Study of NGGT002 in cPKU Adult Subjects
Sponsor:
NGGT (Suzhou) Biotechnology Co., Ltd.
Organization:
Study Overview
Official Title:
A Phase I/II Study for the Safety and Efficacy of Intravenous Infusion With NGGT002 in Adults Patients With Classic Phenylketonuria
Status:
RECRUITING
Status Verified Date:
2024-11
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:
None
Brief Summary:
This is a Phase 1/2, open-label, multiple-center, dose escalation and cohort expansion study to evaluate the safety and efficacy of NGGT002 in adult subjects with classic Phenylketonuria (PKU). NGGT002 is a rAAV8 based vector carrying a functional copy of the human PAH gene.
Participants will receive a single administration of NGGT002 and will be followed for safety and efficacy for 5 years.
Participants will receive a single administration of NGGT002 and will be followed for safety and efficacy for 5 years.
Detailed Description:
This study will evaluate the safety and efficacy of NGGT002 gene therapy with three dose cohorts in adult subjects with a diagnosis of classic PKU, a condition characterized by severe PAH deficiency with no residual enzyme activity. NGGT002 will be administered through intravenous infusion.
Study Oversight
Has Oversight DMC:
False
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?: