Ignite Creation Date:
2024-05-06 @ 7:35 PM
Last Modification Date:
2024-10-26 @ 3:09 PM
Study NCT ID:
NCT06061614
Status:
RECRUITING
Last Update Posted:
2024-02-07
First Post:
2023-09-25
Brief Title:
Safety and Efficacy Study of NGGT002 in PKU Adult Subjects
Sponsor:
First Affiliated Hospital Bengbu Medical College
Organization:
First Affiliated Hospital Bengbu Medical College
Study Overview
Official Title:
A Clinical Study for the Safety and Efficacy of IV Infusion of NGGT002 in the Treatment of Phenylketonuria
Status:
RECRUITING
Status Verified Date:
2024-02
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:
None
Brief Summary:
This investigator initiated trial is an open-label dose escalation study to evaluate the safety tolerability and efficacy of NGGT002 an AAV derived investigational gene therapy product expressing human PAH enzyme in adult Phenylketonuria PKU subjects with PAH deficiency All 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 diagnosis of PKU characterized by PAH deficiency with confirmed PAH gene mutations NGGT002 will be administered through intravenous infusion Dose escalation will be initiated from the low dose of NGGT002 After evaluating the safety and efficacy data a decision will be made regarding the cohort expansion or the escalation to the next dose level The same process will be followed for subsequent dose cohorts
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