Safety and Efficacy Study of NGGT002 in cPKU Adult Subjects

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.

Timeline

Start

2024-07-25

Primary completion

2026-07-30

Completion

2031-07-30

Drugs

Indications