drugset / Trial / NCT03485677
Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3
Non-randomizedSequentialOpen-labelTreatment
Summary
Primary Objective: Evaluated the safety and pharmacokinetics of eliglustat in pediatric participants (≥2 to \<18 years old). Secondary Objective: Evaluated the efficacy of eliglustat and quality of life in pediatric participants (≥2 to \<18 years old).
Timeline
- Start
- 2018-04-11
- Primary completion
- 2025-12-26
- Completion
- 2025-12-26
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Eliglustat GZ385660 | Unknown | — | Oral |
| Subject | Imiglucerase | Protein / enzyme biologic | — | Intravenous |