drugset / Trial / NCT07318909
To Evaluate the Safety and Efficacy of GS1168 Injection in Adult Phenylketonuria
NaSingle-groupOpen-labelTreatment
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.
Timeline
- Start
- 2025-12-31
- Primary completion
- 2027-08-30
- Completion
- 2031-08-30
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | GS1168 injection | Gene therapy (AAV / viral vector) | — | Intravenous |