Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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EUCTR |
Last refreshed on:
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20 August 2018 |
Main ID: |
EUCTR2016-000301-37-ES |
Date of registration:
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06/07/2018 |
Prospective Registration:
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No |
Primary sponsor: |
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Public title:
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Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Disease (GD) Type 1 and Type 3
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Scientific title:
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Open label, Two Cohort (with and without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients with Gaucher Disease Type 1 and Type 3 - ELIKIDS |
Date of first enrolment:
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21/06/2018 |
Target sample size:
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120 |
Recruitment status: |
Authorised-recruitment may be ongoing or finished |
URL:
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https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-000301-37 |
Study type:
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Interventional clinical trial of medicinal product |
Study design:
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Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: Placebo: Other: Number of treatment arms in the trial: 2
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Phase:
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Human pharmacology (Phase I): no
Therapeutic exploratory (Phase II): no
Therapeutic confirmatory - (Phase III): yes
Therapeutic use (Phase IV): no
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Countries of recruitment
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Argentina
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Canada
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Egypt
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France
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Italy
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Netherlands
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Russian Federation
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Spain
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Sweden
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Turkey
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United Kingdom
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Contacts
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Name:
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Unidad Estudios Clínicos
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Address:
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c/ Josep Pla nº2, 4ª planta
08019
Barcelona
Spain |
Telephone:
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93 485 94 00 |
Email:
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ES-unidadestudiosclinicos@sanofi.com |
Affiliation:
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sanofi-aventis, s.a. |
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Name:
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Unidad Estudios Clínicos
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Address:
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c/ Josep Pla nº2, 4ª planta
08019
Barcelona
Spain |
Telephone:
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93 485 94 00 |
Email:
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ES-unidadestudiosclinicos@sanofi.com |
Affiliation:
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sanofi-aventis, s.a. |
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Key inclusion & exclusion criteria
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Inclusion criteria: -The patient is 6 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 6 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 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 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. Are the trial subjects under 18? yes Number of subjects for this age range: 120 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
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.
Age minimum:
Age maximum:
Gender:
Female: yes Male: yes
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Health Condition(s) or Problem(s) studied
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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
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Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
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Intervention(s)
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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: ELIGLUSTAT TARTRATE Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 84-
Trade Name: Cerezyme Product Name: Imiglucerase Pharmaceutical Form: Powder for concentrate for solution for infusion INN or Proposed INN: IMIGLUCERASE CAS Number: 154248-97-2 Other descriptive name: Cerezyme Concentration unit: U unit(s) Concentration type: equal Concentration number: 400-
Product Name: Eliglustat Product Code: GZ385660 Pharmaceutical Form: Capsule, hard INN or Proposed INN: Eliglustat Current Sponsor code: GZ385660 Other descriptive name: ELIGLUSTAT TARTRATE Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 21-
Product Name: Eliglustat Product Code: GZ385660 Pharmaceutical Form: Capsule, hard INN or Proposed INN: Eliglustat Current Sponsor code: GZ385660 Other descriptive name: ELIGLUSTAT TARTRATE Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 42-
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Primary Outcome(s)
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Main Objective: Evaluate the safety and pharmacokinetics of eliglustat in pediatric patients (=6 to <18 years old).
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Primary end point(s): 1- Maximum concentration (Cmax) of eliglustat in plasma. 2- Area under the plasma eliglustat concentration-time curve (AUC) 3- Number of adverse events in pediatric patients
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Secondary Objective: Evaluate the efficacy of eliglustat and quality of life in pediatric patients (=6 to <18 years old).
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Timepoint(s) of evaluation of this end point: 1- Maximum concentration (Cmax) of eliglustat in plasma : Weeks 2, 13, 26 and 52 2- Area under the plasma eliglustat concentration-time curve (AUC) : Weeks 2 and 52 3- Number of adverse events in pediatric patients : Up to Week 104
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Secondary Outcome(s)
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Timepoint(s) of evaluation of this end point: Baseline and Week 52
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Secondary end point(s): 1- Absolute change from baseline for hemoglobin (g/dL) (Cohort 1 patients) 2- Percent change from baseline for platelet count (Cohort 1 patients) 3- Percent change from baseline for liver volume (Cohort 1 patients) 4- Percent change from baseline for spleen volume (Cohort 1 patients) 5- Proportion of patients with improvement in pulmonary disease (Cohort 2 patients) 6- Proportion of patients with improvement in bone disease (Cohort 2 patients) 7- Proportion of patients with improvement in thrombocytopenia (Cohort 2 patients) 8- Health-related quality of life will be measured by the Pediatric Quality of Life InventoryTM (PedsQLTM) questionnaires.
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Secondary ID(s)
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EFC13738
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2016-000301-37-NL
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Source(s) of Monetary Support
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Genzyme Corporation
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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