Gaucher's disease type I; Gaucher's disease type III
Conditions
Brief summary
Assessment of pharmacokinetic (PK) parameter of eliglustat: Cmax ; Maximum concentration (Cmax) of eliglustat in plasma, Assessment of PK parameter of eliglustat: AUC ; Area under the plasma eliglustat concentration-time curve (AUC), Adverse Events; Number of adverse events in pediatric patients
Detailed description
Change in hemoglobin level; Absolute change from baseline for hemoglobin (g/dL) (Cohort 1 patients), Change in platelet count; Percent change from baseline for platelet count (Cohort 1 patients), Change in liver volume; Percent change from baseline for liver volume (Cohort 1 patients), Change in spleen volume; Percent change from baseline for spleen volume (Cohort 1 patients), Pulmonary disease improvement; Proportion of patients with improvement in pulmonary disease (Cohort 2 patients), Bone disease improvement; Proportion of patients with improvement in bone disease (Cohort 2 patients), Thrombocytopenia; Proportion of patients with improvement in thrombocytopenia (Cohort 2 patients), Quality of Life; Health-related quality of life will be measured by the Pediatric Quality of Life InventoryTM (PedsQLTM) questionnaires
Interventions
Sponsors
Eligibility
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Assessment of pharmacokinetic (PK) parameter of eliglustat: Cmax ; Maximum concentration (Cmax) of eliglustat in plasma, Assessment of PK parameter of eliglustat: AUC ; Area under the plasma eliglustat concentration-time curve (AUC), Adverse Events; Number of adverse events in pediatric patients | — |
Secondary
| Measure | Time frame |
|---|---|
| Change in hemoglobin level; Absolute change from baseline for hemoglobin (g/dL) (Cohort 1 patients), Change in platelet count; Percent change from baseline for platelet count (Cohort 1 patients), Change in liver volume; Percent change from baseline for liver volume (Cohort 1 patients), Change in spleen volume; Percent change from baseline for spleen volume (Cohort 1 patients), Pulmonary disease improvement; Proportion of patients with improvement in pulmonary disease (Cohort 2 patients), Bone disease improvement; Proportion of patients with improvement in bone disease (Cohort 2 patients), Thrombocytopenia; Proportion of patients with improvement in thrombocytopenia (Cohort 2 patients), Quality of Life; Health-related quality of life will be measured by the Pediatric Quality of Life InventoryTM (PedsQLTM) questionnaires | — |
Countries
France, Italy, Spain