Actively Recruiting
A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients With Wilson Disease
Led by Chaohui Yu · Updated on 2026-01-23
18
Participants Needed
1
Research Sites
287 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Wilson's disease (WD), also known as Wilson's disease, is a rare autosomal recessive metabolic disorder caused by a mutation of the copper transport ATPase β (ATP7B) gene located on the long arm of chromosome 13 (13q14.3). This leads to accumulation of copper ions in multiple organs such as liver, brain and kidney, resulting in organ involvement. In this study, LY-M003 Injection is a gene therapy products with rAAV8 vector. After a single intravenous infusion, LY-M003 can be transduced to the target organ of liver and express the ATP7B in hepatocytese.
CONDITIONS
Official 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 provide written informed consent voluntarily
- Diagnosed with Wilson Disease
- Laboratory confirmation of biallelic mutations in the ATP7B gene
- Have received standard treatment for Wilson Disease for at least 6 months before screening
- Have restricted high copper foods for at least 6 months before screening and agree to maintain this during the study
- Willing to refrain from donating blood, organs, tissues, or cells during study participation
- Negative pregnancy test for women who can become pregnant
- Willing to use effective contraception from screening until 6 months after study ends and no plans to donate sperm or eggs
You will not qualify if you...
- AAV8 neutralizing antibody titer greater than 1:10
- Active gastrointestinal bleeding within the last 3 months
- Decompensated cirrhosis or advanced liver disease with complications
- Other liver diseases like immune hepatitis or alcoholic liver disease
- Severe hypersplenism requiring splenectomy
- Model for End-Stage Liver Disease (MELD) Score over 13
- Other copper metabolism disorders
- Noncompliance with copper chelators or zinc agents within 6 months prior to screening
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels 5 times above normal in treated patients
- Severe central nervous system symptoms needing urgent hospitalization
- Hemoglobin below 90 g/L
- History of seizures or other diseases affecting study compliance within 6 months
- Positive tests for hepatitis B, hepatitis C, HIV, or syphilis
- End-stage kidney disease requiring dialysis or creatinine clearance below 60 mL/min
- Severe hyperlipidemia (triglycerides over 1000 mg/dL)
- Received or plans for bone marrow, stem cell, or major organ transplant
- Serious cardiovascular disease or heart failure class 3 or above
- Uncontrolled diseases or infections
- Allergy to any component of LY-M003
- Previous gene or cell therapy
- Use of systemic immunosuppressants or steroids within 3 months prior to dosing (except protocol-specified prophylaxis)
- Cancer history within 5 years except certain fully treated cancers
- Vaccinated with live attenuated vaccine within 4 months prior or plans to receive during trial
- Received other investigational drugs or devices within 28 days or 5 half-lives prior to screening
- Pregnant or breastfeeding women
- Other investigator-determined reasons making participation inappropriate
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
CONTACT
Y
Yi Chen, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
5
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