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 .
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
15
GC310 is an adeno-associated virus 5 (AAV5) vector delivering a functional copy of the truncated human ATP7B gene
Peking Union Medical College
Beijing, China
Incidence of adverse events after GC310 administration
Time frame: within 12 weeks
Incidence of dose-limiting toxicity (DLT) events after GC310 administration;
Time frame: within 4 weeks
Change from baseline in serum ceruloplasmin (CP) concentration after GC310 administration;
Time frame: 52 weeks
Change from baseline in 24-hour urinary copper excretion after GC310 administration.
Time frame: 52 weeks
Change from baseline in the urinary copper-to-creatinine ratio
Time frame: 52 weeks
Change from baseline in ALT and AST levels
Time frame: 52 weeks
Change from baseline in hepatic imaging findings
Time frame: 52 weeks
Change from baseline in Kayser-Fleischer (K-F) rings observed by slit-lamp examination
Time frame: 52 weeks
Evaluation of adverse-event incidence
Time frame: 52 weeks
Serum anti-AAV5 and anti-ATP7B antibody levels
Time frame: 52 weeks
Change in blood GC310 vector genome copy number
Time frame: 52 weeks
AAV shedding
Time frame: 52 weeks
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