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

Sanofi-aventis0 个研究点目标入组 2 人开始时间: 待定最近更新: 2024年4月15日

适应症

试验速览

阶段: 3 期
状态: 撤回
发起方: Sanofi-aventis
入组人数: 2

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

入排标准

年龄范围: 2 至 17（—）

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 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.
•-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.

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

Sanofi-aventis

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