Ignite Creation Date:
2024-05-06 @ 8:19 PM
Last Modification Date:
2024-10-26 @ 3:25 PM
Study NCT ID:
NCT06332807
Status:
RECRUITING
Last Update Posted:
2024-06-17
First Post:
2024-02-07
Brief Title:
AAV Gene Therapy Clinical Study in Adult Classic PKU PHEdom
Sponsor:
NGGT INC
Organization:
NGGT INC
Study Overview
Official Title:
An Open-Label Multiple-Center Phase III Dose Escalation Study for the Safety and Efficacy of NGGT002 in Adults With Classic Phenylketonuria
Status:
RECRUITING
Status Verified Date:
2024-09
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 12 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 an 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 two dose cohorts in adult subjects with diagnosis of classic PKU a condition characterized by severe PAH deficiency with no residual enzyme activity NGGT002 will be administered through intravenous infusion In Part 1 of the study subjects will receive NGGT002 at the low dose Dosing of the first 3 subjects will be staggered Following evaluation of data from the first 3 subjects a decision can be made to either escalate to the high dose level or expand the low dose cohort with additional 3 subjects Upon completion of Part 1 study based on the evaluation of and safety and efficacy the study may be stopped or proceed to Part 2 In Part 2 the same process will be conducted with 3 -6 subjects dosed at the high dose
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
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