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

Genzyme Corporation0 个研究点目标入组 120 人开始时间: 2018年6月28日最近更新: 2019年4月30日

试验速览

阶段: 1 期
状态: 进行中（未招募）
发起方: Genzyme Corporation
入组人数: 120

研究概览入排标准研究者记录与标识符相似试验

研究概览

简要总结

暂无简介。

研究设计

研究类型: Interventional clinical trial of medicinal product

入排标准

入选标准

•-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
•-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.
•-Symptomatic bone disease characterized by pathological fracture, osteonecrosis,
•osteopenia/osteoporosis, or bone crisis occurring in the 12 months prior to enrollment.
•-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

排除标准

•-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.

研究者

发起方

Genzyme Corporation

相似试验

进行中（未招募）

1 期

Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Disease (GD) Type 1 and Type 3Congenital, hereditary and neonatal diseasesMedDRA version: 20.0Level: PTClassification code 10075697Term: Gaucher's disease type ISystem Organ Class: 10010331 - Congenital, familial and genetic disordersMedDRA version: 20.0Level: PTClassification code 10075699Term: Gaucher's disease type IIISystem Organ Class: 10010331 - Congenital, familial and genetic disorders

EUCTR2016-000301-37-SEGenzyme Corporation120

进行中（未招募）

1 期

Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Disease (GD) Type 1 and Type 3Congenital, hereditary and neonatal diseasesMedDRA version: 20.0 Level: PT Classification code 10075697 Term: Gaucher's disease type I System Organ Class: 10010331 - Congenital, familial and genetic disordersMedDRA version: 20.0 Level: PT Classification code 10075699 Term: Gaucher's disease type III System Organ Class: 10010331 - Congenital, familial and genetic disorders

EUCTR2016-000301-37-GBGenzyme Corporation120

进行中（未招募）

1 期

EUCTR2016-000301-37-ITGenzyme Corporation120

进行中（未招募）

1 期

EUCTR2016-000301-37-ESGenzyme Corporation120

撤回

3 期

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 3Gaucher diseaselysosomal storage diseaseerfelijke lysosomale stofwisselingsziekte (Gaucher)10027424

NL-OMON48873Sanofi-aventis2