Last updated on December 2019

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


Brief description of study

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 Study 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) and a long-term treatment period (Week 53 to Week 104). After study completion, patients will be encouraged to enroll in the International Collaborative Gaucher Group (ICGG) Gaucher Registry.

Clinical Study Identifier: NCT03485677

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