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

NCT06650319 ยท Status: RECRUITING ยท Phase: EARLY_PHASE1 ยท Type: INTERVENTIONAL ยท Enrollment: 18

Last updated 2026-01-23

No results posted yet for this study

Summary

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 ฮฒ (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

  • Wilson Disease

Interventions

GENETIC

LY-M003

A single peripheral intravenous (IV) infusion of LY-M003

Sponsors & Collaborators

  • Chaohui Yu

    lead OTHER

Principal Investigators

  • Chaohui Yu, PhD ยท First Affiliated Hospital of Zhejiang University

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
10 Years
Max Age
60 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-09-24
Primary Completion
2026-12-30
Completion
2030-03-30

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06650319 on ClinicalTrials.gov