Clinical Trial

Efficacy and Safety of Eliglustat in Chinese Pediatric Patients With Gaucher Disease Type 1 and Type 3

Unknown Phase 2
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Record status
This record was last updated July 26, 2024 (before its estimated July 31, 2025 completion). Its status may not reflect the trial's current state.
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.
Protocol Amendment History 1 change
critical Trial status changed: Not Yet Recruiting → Unknown 2026-08-02
Trial Details
NCT Number NCT06523517
Lead Sponsor Peking Union Medical College Hospital
Conditions Gaucher Disease
Enrollment 5 participants
Start Date 2024-08-01
Primary Completion 2025-07-31 (estimated)
Study Completion 2025-07-31 (estimated)
Updated on ClinicalTrials.gov 2024-07-26