Actively Recruiting
Prospective, Single-center, Open-label, Single-arm, Single-dose Clinical Study to Evaluate the Safety, Tolerability and Efficacy of LY-M003 Injection in Adult and Pediatric Patients With Wilson Disease
Led by Chaohui Yu · Updated on 2026-01-23
18
Participants Needed
1
Research Sites
169 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Wilson's disease is a rare inherited disorder caused by mutations in the ATP7B gene, leading to copper buildup in organs like the liver, brain, and kidneys. Researchers are evaluating LY-M003 Injection, a gene therapy using an rAAV8 vector, to deliver the ATP7B gene to liver cells. This study aims to assess the safety, tolerability, effectiveness, immune response, and how the body processes this therapy in patients with Wilson disease. The study uses a single intravenous infusion of LY-M003 at one of four dose levels, ranging from 1.0 to 6.0 x 10^13 vector genomes per kilogram. There are adult and pediatric groups, with dose escalation decisions based on safety and effectiveness data. Adult participants receive one of four dose levels, while pediatric dosing is determined after reviewing adult results. The study has a main phase and a long-term follow-up phase to monitor outcomes. Participants will be followed for 52 weeks after receiving the infusion. During this time, researchers will monitor adverse events, dose-limiting toxicities, changes in copper-related lab tests, liver function, neurological and psychiatric symptoms, and liver stiffness. They will also track reductions in standard Wilson disease medications. This comprehensive monitoring includes clinical assessments and laboratory tests to evaluate how participants respond to LY-M003 therapy over time.
CONDITIONS
Brief Title
A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients With Wilson Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Able to understand the study and voluntarily consent to participate
- Diagnosed with Wilson Disease
- Confirmed biallelic mutations in the ATP7B gene
- Treated with standard Wilson Disease medications for at least 6 months before screening
- Following a low-copper diet for at least 6 months before screening and willing to continue
- Willing to avoid donating blood, organs, tissues, or cells during the study
- Negative pregnancy test for women of childbearing potential
- Willing to use effective contraception and have no plans for pregnancy or donation of sperm or ova during and 6 months after the study
You will not qualify if you...
- AAV8 neutralizing antibody titer greater than 1:10
- Active gastrointestinal bleeding in the past 3 months
- Decompensated cirrhosis or advanced liver disease
- Other liver diseases such as immune hepatitis or alcoholic liver disease
- Severe hypersplenism requiring splenectomy
- MELD score greater than 13
- Other copper metabolism disorders
- Noncompliance with copper chelators or zinc agents in past 6 months
- ALT or AST levels 5 times above normal in treated patients
- Severe central nervous system symptoms needing hospitalization
- Hemoglobin less than 90 g/L
- Recent epileptic seizures or diseases affecting study compliance
- Positive for hepatitis B, C, HIV, or syphilis antibodies
- End-stage renal disease or creatinine clearance below 60 mL/min
- Severe hyperlipidemia (triglycerides over 1000 mg/dL)
- Prior or planned major organ or stem cell transplantation
- Serious cardiovascular disease
- Uncontrolled infections or diseases
- Allergy to LY-M003 components
- Previous gene or cell therapy
- Recent immunosuppressive or steroid therapy
- History of cancer within 5 years except certain types
- Recent or planned live attenuated vaccinations
- Recent participation in other investigational drug/device trials
- Pregnant or breastfeeding women
- Any other condition deemed unsuitable by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 screening and enrollment visit (in-person)
Duration - Single day
Participants receive a single peripheral intravenous (IV) infusion of LY-M003 at one of four dose levels depending on cohort and safety data.
1 infusion visit (in-person)
Duration - 52 weeks
Participants are monitored for safety, tolerability, and efficacy outcomes for up to 52 weeks after the LY-M003 injection.
Multiple follow-up visits over 52 weeks
Trial Site Locations
Total: 1 location
1
First Affiliated Hospital of Zhejiang University
Hangzhou, Zhejiang, China, 312000
Actively Recruiting
Research Team
C
Chaohui Yu, PhD
Y
Yi Chen, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
5
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here