A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients With Wilson Disease

ConditionWilson Disease

Trial statusRecruiting

Trial phaseEarly Phase 1

Trial typeInterventional

Biological sexAll

Age10-60

SponsorChaohui Yu

About this trial

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.

Eligibility criteria

Qualifiers

The subject must be able to fully understood the purpose, nature, method, and possible adverse effects of the study, must be able to voluntarily participate in the study and voluntarily able to provide the written informed consent form (ICF).

Patients diagnosed with Wilson Disease .

Wilson Disease (WD) patients confirmed by laboratory tests to have biallelic mutations in the ATP7B gene.

Subjects must be treatment-experienced to WD who have received standard treatment (eg, D-penicillamine or zinc acetate) for at least 6 months prior to the screening period.

Disqualifiers

AAV8 neutralizing antibody titer > 1:10 .

Active gastrointestinal bleeding within the past 3 months.

Decompensated cirrhosis or advanced hepatic disease, manifested as portal hypertension, ascites, splenomegaly, esophageal varices, hepatic encephalopathy, etc.

Subjects with other liver diseases as determined by the investigator, such as immune hepatitis, alcoholic liver disease, primary biliary cholangitis, primary sclerosing cholangitis, and/or drug or toxic liver disease

Trial design

Treatments tested in this trial

LY-M003

Treatment groups

18 Participants

are divided into 5 treatment groups

Sponsors and collaborators

Chaohui Yu

Lead sponsor

First Affiliated Hospital of Zhejiang University

Sponsor institution

Source ClinicalTrials.gov