Not Yet RecruitingPhase 1/2

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

China15 participantsStarted 2025-10

Plain-language 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 .

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Exclusion criteria

✕. 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.

What they're measuring

Incidence of adverse events after GC310 administration

Timeframe: within 12 weeks

Incidence of dose-limiting toxicity (DLT) events after GC310 administration;

Timeframe: within 4 weeks

Change from baseline in serum ceruloplasmin (CP) concentration after GC310 administration;

Timeframe: 52 weeks

Change from baseline in 24-hour urinary copper excretion after GC310 administration.

Timeframe: 52 weeks

Trial details

NCT IDNCT07173933

SponsorGeneCradle Inc

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2027-04

Contact for this trial

GeneCradle, Inc China

86-13501380583 ind@bj-genecradle.com

View on ClinicalTrials.gov More Wilson Disease trials

Plain-language summary

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Exclusion criteria

✕. 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.

What they're measuring

Incidence of adverse events after GC310 administration

Timeframe: within 12 weeks

Incidence of dose-limiting toxicity (DLT) events after GC310 administration;

Timeframe: within 4 weeks

Change from baseline in serum ceruloplasmin (CP) concentration after GC310 administration;

Timeframe: 52 weeks

Change from baseline in 24-hour urinary copper excretion after GC310 administration.

Timeframe: 52 weeks