NCT06523517

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

A Single-center, Single-arm, Prospective Clinical Study to Evaluate the Efficacy and Safety of Eliglustat in Chinese Pediatric Patients (≥12 to <18 Years Old) With Gaucher Disease Type 1 and Type 3

• Status: Not Yet Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 5 (estimated)
• Sponsor: Peking Union Medical College Hospital · Academic / Other
• Sex: All
• Age: 12 Years – 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Gaucher Disease

Interventions

Type	Name	Description
DRUG	Eliglustat Tartrate Capsules	The initial dose is 42 mg taken orally twice a day. After 2 weeks of treatment, if the blood trough concentration is less than 5 ng/mL, the dose will be increased to 84 mg taken orally twice daily.

Timeline

• Start date: 2024-08-01
• Primary completion: 2025-07-31
• Completion: 2025-07-31
• First posted: 2024-07-26
• Last updated: 2024-07-26

Locations

1 site across 1 country: China