A Phase III, Multicenter, Randomized, Double-Blind Trial to Assess the Safety and Efficacy 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
- 32
- 试验地点
- 11
- 主要终点
- Change From Baseline in Spleen Volume Measured by MRI.
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 the second trial to utilize a recombinant active form of lysosomal enzyme, glucocerebrosidase, (human prGCD) which is expressed and purified in a bioreactor system from transformed carrot plant root cell line.
详细描述
This will be a multi-center, randomized, double-blind, parallel group, dose-ranging trial to assess the safety and efficacy of prGCD in 30 untreated patients with Gaucher disease. Patients will receive IV infusion of prGCD every two weeks at the selected medical center. The duration of the study will be nine months. At the end of the 9-month treatment period (20 visits, 38 weeks) eligible patients will be offered enrollment in an open-label extension study.
There will be two treatment groups, 15 patients in each treatment group.
Treatment Group I: 30 units/kg every 2 weeks. Treatment Group II: 60 units/kg every 2 weeks.
All patients will have pharmacokinetic data collected over approximately 3 hours with frequent blood samples following the first and final doses of prGCD.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- •Males and females, 18 years or older
- •Confirmed enzymatic diagnosis of Gaucher disease
- •Splenomegaly defined as greater than eight times the expected volume (measured volume divided by estimated volume (0.2% of body weight)] as determined by MRI volumetric analysis
- •Female patients of child-bearing potential who agree to use a medically acceptable method of contraception
- •Thrombocytopenia (defined as platelet counts below the lower limit of normal) and/or anemia (defined by hemoglobin level at least 1 g/dL below normal range according to sex and age).
- •Patients who have not received ERT in the past or patients whoc have not received ERT in the past 12 months and have a negative anti-glucocerebrosidase antibody test.
- •Patients who have not received substrate reduction therapy (SRT) in the past 12 months.
- •Ability to provide a written informed consent.
排除标准
- •Currently taking another experimental drug for any condition
- •Pregnant or nursing
- •Presence of HIV and/or, HBsAg and/or hepatitis C infections
- •Presence of severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic Gaucher disease.
- •Previous anaphylactoid reaction to Cerezyme® or Ceredase®.
- •History of allergy to carrots.
研究组 & 干预措施
prGCD 30 Units/kg
干预措施: Plant cell expressed recombinant glucocerebrosidase (prGCD) (Drug)
prGCD 60 Units/kg
干预措施: Plant cell expressed recombinant glucocerebrosidase (prGCD) (Drug)
结局指标
主要结局
Change From Baseline in Spleen Volume Measured by MRI.
时间窗: Baseline and 9 months
Calculated as percent change in spleen volume from Baseline to 9 months
次要结局
- Change From Baseline in Liver Volume(Baseline and 9 months)
- Change in Hemoglobin(Baseline and Month 9)
- Change in Platelet Count(Baseline and Month 9)