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 .
Aged ≥ 18 years, sex unrestricted;
Serum ceruloplasmin concentration < ½ × lower limit of normal (LLN);
Willing and able to comply with all study procedures, and has provided written informed consent.
Screening serum anti-AAV5 neutralizing antibody titre > 1:100.
ALT or AST ≥ 5 × ULN, direct bilirubin > 1 × ULN, or albumin < 1 × LLN;
Blood ammonia > 1 × ULN.
Renal impairment (any degree).
Single group
GC310
GeneticGC310 is an adeno-associated virus 5 (AAV5) vector delivering a functional copy of the truncated human ATP7B gene
Incidence of adverse events after GC310 administration
Incidence of dose-limiting toxicity (DLT) events after GC310 administration;
Change from baseline in serum ceruloplasmin (CP) concentration after GC310 administration;
Change from baseline in 24-hour urinary copper excretion after GC310 administration.
Change from baseline in the urinary copper-to-creatinine ratio
Change from baseline in ALT and AST levels
Change from baseline in hepatic imaging findings
Change from baseline in Kayser-Fleischer (K-F) rings observed by slit-lamp examination
Change from baseline in serum ceruloplasmin (CP) activity
Change from baseline in relative exchangeable copper (exchangeable copper/total serum copper)
Change from baseline in brain MRI findings
Change from baseline in liver biopsy findings
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This trial is not recruiting at the moment. You can still explore other options:
