EUCTR2016-000301-37-SE进行中（未招募）1 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 3 - ELIKIDS
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 120
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
- 性别
- All
入选标准
- •-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
- •-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
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