drugset / Trial / NCT07641140
Phase I/II Clinical Study to Evaluate the Safety, Tolerability and Efficacy of LY-M003 Injection in Adult Patients With Wilson's Disease
Non-randomizedSequentialOpen-labelTreatment
Summary
This is a multicenter, open-label, single-arm, single-dose Phase I/II clinical study. It aims to evaluate the safety, tolerability, efficacy, immunogenicity, pharmacodynamic (PD) and pharmacokinetic (PK) profiles of LY-M003 Injection in patients with Wilson's Disease (WD).
Timeline
- Start
- 2026-06-15
- Primary completion
- 2027-12-30
- Completion
- 2032-12-30
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| 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 |