drugset / Trial / NCT06650319
A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients With Wilson Disease
Non-randomizedSingle-groupOpen-labelTreatment
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.
Timeline
- Start
- 2024-09-24
- Primary completion
- 2026-12-30
- Completion
- 2030-03-30
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | LY-M003 | Gene therapy (AAV / viral vector) | 1e+13 vg/kg | Intravenous |
| Subject | LY-M003 | Gene therapy (AAV / viral vector) | 2e+13 vg/kg | Intravenous |
| Subject | LY-M003 | Gene therapy (AAV / viral vector) | 4e+13 vg/kg | Intravenous |
| Subject | LY-M003 | Gene therapy (AAV / viral vector) | 6e+13 vg/kg | Intravenous |