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RECRUITING
NCT06687733
PHASE1/PHASE2

Safety and Efficacy Study of NGGT002 in cPKU Adult Subjects

Sponsor: NGGT (Suzhou) Biotechnology Co., Ltd.

View on ClinicalTrials.gov

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.

Official title: A Phase I/II Study for the Safety and Efficacy of Intravenous Infusion With NGGT002 in Adults Patients With Classic Phenylketonuria

Key Details

Gender

All

Age Range

18 Years - 55 Years

Study Type

INTERVENTIONAL

Enrollment

18

Start Date

2024-07-25

Completion Date

2031-07-30

Last Updated

2024-11-14

Healthy Volunteers

No

Interventions

GENETIC

NGGT002

adeno-associated viral vector with human phenylalanine hydroxylase gene

Locations (2)

First Affiliated Hospital of Bengbu Medical College

Bengbu, Anhui, China

Xinhua Hospital Affifiated to Shanghai Jiao Tong University School of Medicine

Shanghai, China