drugset / Trial / NCT06523517
Efficacy and Safety of Eliglustat in Chinese Pediatric Patients With Gaucher Disease Type 1 and Type 3
NaSingle-groupOpen-labelTreatment
Summary
Primary Objective: Evaluate the efficacy and safety of eliglustat in Chinese pediatric patients (≥12 to \<18 years old) with Gaucher disease type 1 and type 3. Secondary Objective: Evaluate the quality of life in Chinese pediatric patients (≥12 to \<18 years old) with Gaucher disease type 1 and type 3 treated with eliglustat.
Timeline
- Start
- 2024-08-01
- Primary completion
- 2025-07-31
- Completion
- 2025-07-31
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Eliglustat | Small molecule | 42 mg | Oral |
| Subject | Eliglustat | Small molecule | 84 mg | Oral |