NCT06687733

Safety and Efficacy Study of NGGT002 in cPKU Adult Subjects

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

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 18 (estimated)
• Sponsor: NGGT (Suzhou) Biotechnology Co., Ltd. · Industry
• Sex: All
• Age: 18 Years – 55 Years
• Healthy volunteers: Not accepted

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.

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.

Conditions

• Phenylketonurias

Interventions

Type	Name	Description
GENETIC	NGGT002	adeno-associated viral vector with human phenylalanine hydroxylase gene

Timeline

• Start date: 2024-07-25
• Primary completion: 2026-07-30
• Completion: 2031-07-30
• First posted: 2024-11-14
• Last updated: 2024-11-14

Locations

2 sites across 1 country: China