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

NCT03485677 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 57

Last updated 2026-01-14

No results posted yet for this study

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).

Conditions

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

Interventions

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

Sponsors & Collaborators

Principal Investigators

  • Clinical Sciences & Operations · Sanofi

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
2 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-04-11
Primary Completion
2025-12-12
Completion
2025-12-12

Countries

  • Argentina
  • Canada
  • France
  • Italy
  • Japan
  • Russia
  • Spain
  • Sweden
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03485677 on ClinicalTrials.gov