A Multicenter, Double-Blind, Extension Trial of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher Disease
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 45
- 试验地点
- 11
- 主要终点
- Spleen Volume
研究概览
简要总结
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the accumulation of substrate glucocerebroside (GlcCer) in the cells of the monocyte-macrophage system.
This is an extension trial to Study NCT00376168 and NCT00712348.
详细描述
This will be a multi-center, double-blind, parallel group, extension trial to assess the safety and efficacy of prGCD in patients completing NCT00376168. Patients will receive IV infusion of prGCD every two weeks at the selected medical center. The duration of the extension study will be fifteen months. There will be two treatment groups: 30 units/kg every 2 weeks or 60 units/kg every 2 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Successful completion of Protocol PB-06-001
- •The patient signs informed consent
排除标准
- •Currently taking another experimental drug for any condition
- •Presence of severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic Gaucher disease
- •Pregnant or nursing
- •Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the patient's compliance with the requirements of the study
研究组 & 干预措施
Naive 30 Units/kg
Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168)
干预措施: Taliglucerase alfa (Drug)
Naive 60 Units/kg
Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168)
干预措施: Taliglucerase alfa (Drug)
Switchover
Continue taliglucerase alfa treatment from PB-06-002 (NCT00712348)
干预措施: Taliglucerase alfa (Drug)
结局指标
主要结局
Spleen Volume
时间窗: Spleen Volume at Baseline and Months 12, 24, and 36
Spleen volume measured by MRI
次要结局
- Platelet Count(Platelet count at Baseline and Months 12, 24 and 36)
- Hemoglobin(Hemoglobin at Baseline and Months 12, 24 and 36)
- Liver Volume(Liver volume at Baseline and Months 12, 24 and 36)