NCT03485677

Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3

Open Label, Two Cohort (With and Without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients With Gaucher Disease Type 1 and Type 3

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 57 (actual)
• Sponsor: Sanofi · Industry
• Sex: All
• Age: 2 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

Primary Objective: Evaluate the safety and pharmacokinetics of eliglustat in pediatric patients (≥2 to \<18 years old). Secondary Objective: Evaluate the efficacy of eliglustat and quality of life in pediatric patients (≥2 to \<18 years old).

Detailed description

The study will include a screening period of up to 60 days (Day -60 to -1), a primary analysis treatment period (Day 1 to Week 52), a long-term treatment period (Week 53 to Week 104), and an extension period continuing up to Week 364 (for patients who continue to demonstrate the clinical benefit from eliglustat monotherapy at Week 104). After study completion, patients will be encouraged to enroll in the International Collaborative Gaucher Group (ICGG) Gaucher Registry.

Conditions

• Gaucher's Disease Type I
• Gaucher's Disease Type III

Interventions

Type	Name	Description
DRUG	Eliglustat GZ385660	Pharmaceutical form: Capsule, Liquid Route of administration: Oral
DRUG	Imiglucerase GZ437843	Pharmaceutical form: Powder for solution for infusion Route of administration: Intravenous

Timeline

• Start date: 2018-04-11
• Primary completion: 2025-12-12
• Completion: 2025-12-12
• First posted: 2018-04-02
• Last updated: 2026-01-14

Locations

21 sites across 10 countries: Argentina, Canada, France, Italy, Japan, Russia, Spain, Sweden, Turkey (Türkiye), United Kingdom