Status:
NOT_YET_RECRUITING
Phase I/II Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of GC310 Injection in Patients With Wilson's Disease (WD)
Lead Sponsor:
GeneCradle Inc
Conditions:
Wilson Disease
Eligibility:
All Genders
18+ years
Phase:
PHASE1
PHASE2
Brief 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 G...
Eligibility Criteria
Inclusion
- Aged ≥ 18 years, sex unrestricted;
- Definitive diagnosis of Wilson disease (WD) based on:
- (i) or (ii) + (iii) and (iv), or (i) or (ii) + (v); (i) Neurological and/or psychiatric symptoms; (ii) Unexplained liver injury; (iii) Reduced serum ceruloplasmin and/or elevated 24-hour urinary copper; (iv) Positive corneal Kayser-Fleischer (K-F) ring; (v) Biallelic pathogenic ATP7B variants confirmed by segregation analysis and variant pathogenicity assessment;
- Serum ceruloplasmin concentration \< ½ × lower limit of normal (LLN);
- Willing and able to comply with all study procedures, and has provided written informed consent.
Exclusion
- Subjects meeting ANY of the following criteria will be excluded:
- Screening serum anti-AAV5 neutralizing antibody titre \> 1:100.
- Clinically significant laboratory abnormality at screening or baseline:
- ALT or AST ≥ 5 × ULN, direct bilirubin \> 1 × ULN, or albumin \< 1 × LLN;
- Blood ammonia \> 1 × ULN.
- Renal impairment (any degree).
- Current hepatic decompensation or history of hepatic decompensation.
- Liver stiffness measurement (LSM) ≥ 15 kPa by transient elastography at screening.
- History of acute liver failure from any cause.
- Evidence of advanced liver disease defined by either:
- MELD score ≥ 12, or
- Child-Pugh score ≥ 7.
- Severe neuro-psychiatric manifestations that, in the investigator's opinion, could compromise subject safety or interfere with study participation.
- Positive for HIV antibody, hepatitis C antibody, Treponema pallidum antibody, or hepatitis B surface antigen.
- Contraindications to glucocorticoid therapy judged by the investigator (e.g., uncontrolled hypertension, systemic fungal infection, glaucoma, osteoporosis, active tuberculosis).
- Concurrent conditions that may interfere with study conduct or assessment, including significant gastrointestinal, cardiovascular, cerebrovascular, renal, endocrine, haematological, immunological, neurological or psychiatric disorders other than Wilson disease.
- Pregnant or lactating women.
- Women of child-bearing potential or fertile men who plan to conceive within 1 year after dosing or are unwilling to use highly effective contraception.
- Body-mass index ≥ 24 kg/m².
- History of severe hypersensitivity to foods or drugs, including recombinant proteins.
- Vaccination within 2 weeks prior to planned dosing.
- Prior exposure to any gene-therapy product.
- Participation in any other clinical trial (WD-related or not) within 3 months before screening.
- Any other condition or circumstance that, in the opinion of the investigator, renders the subject unsuitable for the study (e.g., poor compliance).
Key Trial Info
Start Date :
October 1 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
October 1 2027
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT07173933
Start Date
October 1 2025
End Date
October 1 2027
Last Update
September 15 2025
Active Locations (1)
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1
Peking Union Medical College
Beijing, China, 100005