Congenital, hereditary and neonatal diseases MedDRA version: 20.0 Level: PT Classification code 10075697 Term: Gaucher's disease type I System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0 Level: PT Classification code 10075699 Term: Gaucher's disease type III System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Cerdelga
Product Name: Eliglustat
Product Code: GZ385660
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Eliglustat
Current Sponsor code: GZ385660
Other descriptive name: ELIGLUSTA
Sponsors
Genzyme Corporation
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: -The patient is 2 to =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: -Substrate reduction therapy for GD within 6 months prior to enrollment -Partial or total splenectomy if performed within 2 years prior to enrollment -The patient is transfusion dependent, a history of esophageal varices or liver infarction, elevated liver enzymes, significant congenital cardiac defect, coronary artery disease or left sided heart failure; clinically significant arrhythmias or conduction defect such as Type 2 second degree or third degree atrioventricular (AV) block, complete bundle branch block, prolonged QTc interval, or sustained ventricular tachycardia (VT). -The patient has any clinically significant disease other than GD. -The patient has neurological symptoms other than oculomotor apraxia at study entry. -The patient has received an investigational product within 30 days prior to enrollment. -The patient is unable to receive treatment with imiglucerase due to a known hypersensitivity or is unwilling to receive imiglucerase treatment every 2 weeks. -The patient has a known hereditary galactose intolerance, Lapp lactase deficiency or glucose galactose malabsorption, or is a CYP2D6 ultra-rapid metabolizer or indeterminate metabolizer.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main 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).;Primary end point(s): 1 - Assessment of pharmacokinetic (PK) parameter of eliglustat: Cmax ; Maximum concentration (Cmax) of eliglustat in plasma. 2 - Assessment of PK parameter of eliglustat: AUC ; Area under the plasma eliglustat concentration-time curve (AUC) 3 - Adverse Events ; Number of adverse events in pediatric patients ;Timepoint(s) of evaluation of this end point: 1 : Weeks 2, 13, 26 and 52 2 : Weeks 2 and 52 3 : Up to Week 364 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1 : Change in hemoglobin level ; Absolute change from baseline for hemoglobin (g/dL) (Cohort 1 patients) 2 : Change in platelet count ; Percent change from baseline for platelet count (Cohort 1 patients) 3 : Change in liver volume ; Percent change from baseline for liver volume (Cohort 1 patients) 4 : Change in spleen volume ; Percent change from baseline for spleen volume (Cohort 1 patients) 5 : Pulmonary disease improvement ; Proportion of patients with improvement in pulmonary disease (Cohort 2 patients) 6 : Bone disease improvement ; Proportion of patients with improvement in bone disease (Cohort 2 patients) 7 : Thrombocytopenia ; Proportion of patients with improvement in thrombocytopenia (Cohort 2 patients) 8 : Quality of Life ; Health-related quality of life will be measured by the Pediatric Quality of Life InventoryTM (PedsQLTM) questionnaires. ;Timepoint(s) of evaluation of this end point: 1, 2, 3, 4, 5, 6, 7, 8 : Baseline and Week 52 | — |
Countries
Argentina, Canada, Egypt, France, Italy, Japan, Netherlands, Russian Federation, Spain, Sweden, Taiwan, Turkey, United Kingdom
Contacts
Public ContactClinical Study Unit
Sanofi AB
Outcome results
None listed