drugset / Trial / NCT07173933
Phase I/II Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of GC310 Injection in Patients With Wilson's Disease (WD)
Non-randomizedSingle-groupOpen-labelTreatment
Summary
The goal of this clinical trial is to learn if GC310 (AAV5-ATP7B) gene therapy can treat Wilson's Disease (WD) in patients over the age of 18 years old. The main questions it aims to answer are: Is GC310 safe and tolerable to WD patients? What is the recommended phase II dose (RP2D)? What is the change from baseline in 24-hour urinary copper concentration after 52 weeks of administration? Participants will be administrated GC310 intravenously and be followed up for 52 weeks to observe drug safety, tolerability and efficacy .
Timeline
- Start
- 2025-10
- Primary completion
- 2027-04
- Completion
- 2027-10
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | GC310 | Gene therapy (AAV / viral vector) | 3e+13 vg/kg | Intravenous |
| Subject | GC310 | Gene therapy (AAV / viral vector) | 6e+13 vg/kg | Intravenous |