drugset / Trial / NCT06528080
A Clinical Study for the Treatment of Pediatric and Adolescent Patients With Type 1 Gaucher Disease
Non-randomizedSingle-groupOpen-labelTreatment
Summary
The purpose of this study was to evaluate the safety, tolerability, efficacy, immunogenicity, PD and PK characteristics of LY-M001 injection in children with GD1 aged 6 years ≤ age \< 18 years. This study mainly includes the main study stage and the long-term follow-up study stage.
Timeline
- Start
- 2024-08-01
- Primary completion
- 2027-05-30
- Completion
- 2030-05-30
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | LY-M001 | Gene therapy (AAV / viral vector) | 1e+13 vg/kg | Intravenous |