Status:
ACTIVE_NOT_RECRUITING
Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3
Lead Sponsor:
Sanofi
Conditions:
Gaucher's Disease Type I
Gaucher's Disease Type III
Eligibility:
All Genders
2-17 years
Phase:
PHASE3
Brief 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 ...
Detailed Description
The study will include a screening period of up to 60 days (Day -60 to -1), a primary analysis treatment period (Day 1 to Week 52), a long-term treatment period (Week 53 to Week 104), and an extension...
Eligibility Criteria
Inclusion
- Inclusion criteria :
- The patient is 2 to \<18 years old at the time of informed consent.
- Male and female patients with a clinical diagnosis of Gaucher disease (GD) type 1 or type 3 with documented deficiency of acid beta-glucosidase activity by enzyme assay and glucocerebrosidase (GBA) genotype.
- Postmenarchal female patients must have a documented negative pregnancy test prior to enrollment and throughout the study. Patients must be willing to practice true abstinence in line with their preferred and usual lifestyle, or use a medically accepted form of contraception throughout the study.
- Cohort 1 (Eliglustat monotherapy):
- Patients must have been receiving an enzyme replacement therapy (ERT) for a minimum of 24 months at a monthly dose equivalent to 30 U/kg to 130 U/kg of Cerezyme® (imiglucerase) with treatment ongoing at the time of enrollment. Patients must be at pre-specified treatment goals, as defined by:
- Hemoglobin level for ages 2 to \<12 years: ≥11.0 g/dL; for ages 12 to \<18 years: ≥11.0 g/dL for females and ≥12.0 g/dL for males;
- Platelet count ≥100,000/mm3;
- Spleen volume \<10.0 multiples of normal (MN);
- Liver volume \<1.5 MN;
- Absence of GD related pulmonary disease, and severe bone disease, as defined below for Cohort 2.
- Cohort 2 (Eliglustat plus imiglucerase):
- Patients must have been receiving an ERT for a minimum of 36 months at a dose equivalent to at least 60 U/kg of imiglucerase every 2 weeks, or at the maximum dose locally approved, at the time of enrollment with treatment ongoing at the time of enrollment and the dose stable for at least the 6 months preceding enrollment. Patients must have severe clinical manifestations of GD, as defined by the presence of at least one of the following:
- GD related pulmonary disease such as interstitial lung disease (ILD). The diagnosis of ILD must be confirmed by the presence of reticulonodular densities on chest X-ray; AND/OR
- Symptomatic bone disease characterized by pathological fracture, osteonecrosis, osteopenia/osteoporosis, or bone crisis occurring in the 12 months prior to enrollment; AND/OR
- Persistent thrombocytopenia (\<80,000/mm3) related to GD.
- 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.
- The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Exclusion
Key Trial Info
Start Date :
April 11 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 20 2025
Estimated Enrollment :
57 Patients enrolled
Trial Details
Trial ID
NCT03485677
Start Date
April 11 2018
End Date
November 20 2025
Last Update
February 20 2024
Active Locations (21)
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1
Investigational Site Number : 0320001
Capital Federal, Buenos Aires, Argentina, C1425DUC
2
Investigational Site Number : 1240002
Calgary, Alberta, Canada, T3B 6A8
3
Investigational Site Number : 1240003
Vancouver, British Columbia, Canada, V6H 3V4
4
Investigational Site Number : 1240001
Toronto, Ontario, Canada, M5G 1X8