Found 248 clinical trials
Venglustat in Combination With Cerezyme in Adult and Pediatric Patients With Gaucher Disease Type 3
Primary Objectives: Part 1: Cohorts 1, 2 and 3 Evaluate cerebrospinal fluid (CSF) biomarkers in adult Gaucher disease (GD) Type 3 patients that distinguish GD3 from adult Gaucher disease Type 1 (GD1) patients. Screen adult and pediatric GD3 patients who qualify for treatment with venglustat in Parts 2 and 3.
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- 04 Dec, 2020
- 7 locations
Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3
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).
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- 04 Dec, 2020
- 23 locations
Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3
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).
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- 04 Dec, 2020
- 19 locations
