NCT07318909

To Evaluate the Safety and Efficacy of GS1168 Injection in Adult Phenylketonuria

A Clinical Exploration Study of Adeno-Associated Virus Vector Expressing Human Phenylalanine Hydroxylase Gene in Adult Classical Phenylketonuria

• Status: Not Yet Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 9 (estimated)
• Sponsor: Gritgen Therapeutics Co., Ltd. · Industry
• Sex: All
• Age: 18 Years – 55 Years
• Healthy volunteers: Not accepted

Summary

This study is a single-arm, open-label, dose-escalation, exploratory study to evaluate the safety, tolerability, and efficacy of a single administration of GS1168 Injection in Chinese adult phenylketonuria (PKU) with PAH mutation.

Conditions

• Phenylketonuria (PKU)

Interventions

Type	Name	Description
DRUG	GS1168 injection	The dose levels are as follows: 1. A single intravenous administration of GS1168 injection at dose level 1; 2. A single intravenous administration of GS1168 injection at dose level 2; 3. A single intravenous administration of GS1168 injection at dose level 3.

Timeline

• Start date: 2025-12-31
• Primary completion: 2027-08-30
• Completion: 2031-08-30
• First posted: 2026-01-06
• Last updated: 2026-01-06

Locations

1 site across 1 country: China