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Wilson DiseasePhase 1/2NCT07173933

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

A Phase 1/2 study testing GC310 in people with Wilson Disease, run by GeneCradle Inc at 1 site in China.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
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Trial 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… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Wilson Disease
What is being tested?
GC310
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
15 participants
Where
1 siteChina
Study dates
Oct 2025to Oct 2027
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Aged ≥ 18 years, sex unrestricted;
  • Definitive diagnosis of Wilson disease (WD) based on:
  • 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.

Who cannot join

  • 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).
Show all 24Show fewer
  • 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).

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

GeneCradle, Inc China contact

86-13501380583

ind@bj-genecradle.com

China1 site

  • Peking Union Medical College

    Beijing, 100005

    Juan Ding, 18810530992, dingjuan8008@163.com

    Zhengqing Qiu

Other trials in China

About this study

Official title: A Multicenter, Open-label, Single-dose, Dose-escalation Phase I/II Clinical Trial Evaluating the Safety, Tolerability, and Efficacy of GC310 Adeno-associated Virus Injection in the Treatment of Patients With Wilson's Disease (WD)

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 .

Conditions
Wilson Disease
Being tested
GC310
Keywords
gene therapy, Genecradle, AAV5, ATP7B, Wilson Disease
Medical subject headings
Hepatolenticular Degeneration

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • GC310Genetic

    GC310 is an adeno-associated virus 5 (AAV5) vector delivering a functional copy of the truncated human ATP7B gene

Study groups in NCT07173933
Study groupRoleWhat they receive
Cohort 1ExperimentalSingle intravenous administration of GC310 at a dose of 3.0E+13 d.vg/kg
Receives: Genetic: GC310
Cohort 2ExperimentalSingle intravenous administration of GC301 at a dose of 6.0E+13 d.vg/kg
Receives: Genetic: GC310

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of adverse events after GC310 administration

    Measured: within 12 weeks

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

    Measured: within 4 weeks

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

    Measured: 52 weeks

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

    Measured: 52 weeks

Secondary outcomes (8)

  1. Change from baseline in the urinary copper-to-creatinine ratio

    Measured: 52 weeks

  2. Change from baseline in ALT and AST levels

    Measured: 52 weeks

  3. Change from baseline in hepatic imaging findings

    Measured: 52 weeks

  4. Change from baseline in Kayser-Fleischer (K-F) rings observed by slit-lamp examination

    Measured: 52 weeks

  5. Evaluation of adverse-event incidence

    Measured: 52 weeks

  6. Serum anti-AAV5 and anti-ATP7B antibody levels

    Measured: 52 weeks

  7. Change in blood GC310 vector genome copy number

    Measured: 52 weeks

  8. AAV shedding

    Measured: 52 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Sep 2025
  2. Study startOct 2025 (estimated)
  3. Primary completionApr 2027 (estimated)
  4. Study completionOct 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
15 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07173933
Sponsor's protocol ID
JLJY-GC310-WD-001
Status last verified
Sep 2025
First submitted
8 Sep 2025
Last updated
15 Sep 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing GC310 in people with Wilson Disease, run by GeneCradle Inc at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 24 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: opening soon, as of the sponsor's last update on 15 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact GeneCradle, Inc China (86-13501380583, ind@bj-genecradle.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of adverse events after GC310 administration (time frame: within 12 weeks). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07173933.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Sep 2025.

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