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

NCT07173933 | Not Yet Recruiting Phase 1 DMC

• 1 other identifier: JLJY-GC310-WD-001
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

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 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

Keywords

gene therapy; Genecradle; AAV5; ATP7B; Wilson Disease

Outcome Measures

Primary Outcomes (4)

• Incidence of adverse events after GC310 administration

  within 12 weeks

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

  within 4 weeks

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

  52 weeks

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

  52 weeks

Secondary Outcomes (8)

• Change from baseline in the urinary copper-to-creatinine ratio

  52 weeks

• Change from baseline in ALT and AST levels

  52 weeks

• Change from baseline in hepatic imaging findings

  52 weeks

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

  52 weeks

• Evaluation of adverse-event incidence

  52 weeks

Other Outcomes (4)

• Change from baseline in serum ceruloplasmin (CP) activity

  52 weeks

• Change from baseline in relative exchangeable copper (exchangeable copper/total serum copper)

  52 weeks

• Change from baseline in brain MRI findings

  52 weeks

Study Arms (2)

Cohort 1

EXPERIMENTAL

Single intravenous administration of GC310 at a dose of 3.0E+13 d.vg/kg

Genetic: GC310

Cohort 2

EXPERIMENTAL

Single intravenous administration of GC301 at a dose of 6.0E+13 d.vg/kg

Genetic: GC310

Interventions

GC310 GENETIC

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

Cohort 1; Cohort 2

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• GeneCradle Inc: lead

Study Sites (1)

Peking Union Medical College

Beijing, 100005, China

Location

MeSH Terms

Conditions

Hepatolenticular Degeneration

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Metal Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

GeneCradle, Inc China

CONTACT

86-13501380583; ind@bj-genecradle.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 8, 2025
• First Posted: September 15, 2025
• Study Start: October 1, 2025
• Primary Completion (Estimated): April 1, 2027
• Study Completion (Estimated): October 1, 2027
• Last Updated: September 15, 2025

Record last verified: 2025-09

Locations

CN (1)