Clinical Trial

Phase I/II Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of GC310 Injection in Patients With Wilson's Disease (WD)

Not Yet Recruiting Phase 1/2
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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 .
Trial Details
NCT Number NCT07173933
Lead Sponsor GeneCradle Inc
Conditions Wilson Disease
Enrollment 15 participants
Start Date 2025-10
Primary Completion 2027-04 (estimated)
Study Completion 2027-10 (estimated)
Updated on ClinicalTrials.gov 2025-09-15