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RECRUITING
NCT06650319
EARLY_PHASE1

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

Sponsor: Chaohui Yu

View on ClinicalTrials.gov

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 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.

Official title: 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

Key Details

Gender

All

Age Range

10 Years - 60 Years

Study Type

INTERVENTIONAL

Enrollment

18

Start Date

2024-09-24

Completion Date

2030-03-30

Last Updated

2026-01-23

Healthy Volunteers

No

Conditions

Interventions

GENETIC

LY-M003

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

Locations (1)

First Affiliated Hospital of Zhejiang University

Hangzhou, Zhejiang, China