Efficacy and Safety of Eliglustat in Chinese Pediatric Patients With Gaucher Disease Type 1 and Type 3
Status:
NOT_YET_RECRUITING
Trial end date:
2025-07-31
Target enrollment:
Participant gender:
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.