NCT06061614

Safety and Efficacy Study of NGGT002 in PKU Adult Subjects

A Clinical Study for the Safety and Efficacy of IV Infusion of NGGT002 in the Treatment of Phenylketonuria

• Status: Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 15 (estimated)
• Sponsor: The First Affiliated Hospital of Bengbu Medical University · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a single-center, open-label, non-randomized, dose escalation study to evaluate the safety, tolerability and efficacy of NGGT002 in adult Phenylketonuria (PKU) subjects. All subjects will receive a single administration of NGGT002 and will be followed for safety and efficacy for 5 years.

Detailed description

This study is designed to evaluate the safety and efficacy of NGGT002 gene therapy in adult subjects diagnosis with PKU due to confirmed PAH gene mutations indicative of PAH deficiency. NGGT002 will be administered via intravenous infusion. The study will begin with Dose Level 1, followed by a stepwise dose escalation. After evaluating the safety and efficacy data, a decision will be made to either expand the current cohort or proceed to the next dose level. The same process will be followed for subsequent dose cohorts.

Conditions

• Phenylketonurias

Interventions

Type	Name	Description
GENETIC	NGGT002 Injection	Dose escalation will proceed sequentially from Dose Level 1 to Dose Level 5 in a single-dose administration of NGGT002.

Timeline

• Start date: 2023-03-30
• Primary completion: 2028-12-30
• Completion: 2028-12-30
• First posted: 2023-09-29
• Last updated: 2026-03-04

Locations

1 site across 1 country: China