A Phase IV, Multicenter, Randomized, Dose Frequency Study of the Safety and Efficacy of Cerezyme® Infusions Every Four Weeks Versus Every Two Weeks in the Maintenance Therapy of Patients With Type 1 Gaucher Disease
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 95
- 试验地点
- 26
- 主要终点
- Number of Participants With Clinical Success at Month 24/Discontinuation
研究概览
简要总结
This is a multicenter, randomized trial to compare the safety and efficacy of two dosing frequencies of Cerezyme® in patients with Gaucher disease who are currently being treated with Cerezyme®.
Approximately 90 patients will be randomized in a 2:1 (q4 : q2) ratio to one of two treatment arms at up to 26 study centers worldwide. Patients will continue to receive the same total 4-week dose that they were receiving prior to study enrollment, however, they will be randomized to receive either their total 4-week dose in two infusions, one infusion every 2 weeks or their total 4-week dose in one infusion every 4 weeks. The randomization scheme will ensure a 2:1 balance between the every 4-week versus every 2-week infusion groups, respectively.
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
- 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
- •The patient must provide written informed consent prior to undergoing any study-related procedures.
- •The patient has a confirmed diagnosis of Gaucher disease with a documented deficiency of glucocerebrosidase by enzyme assay
- •The patient has been genotyped or will have genotyping performed within 3 months of study enrollment.
- •The patient has been treated with Cerezyme for at least 2 years prior to study enrollment.
- •The patient has been on a stable dose of between 20-60 U/kg every 2 weeks for at least 6 months prior to study enrollment.
- •The patient is at least 18 years old.
- •The patient has a hemoglobin value of ≥ 11.0 g/dL for women and ≥ 12.0 g/dL for men and a platelet count of ≥ 100,000 mm^
- •The patient's liver volume is ≤ 1.8 x normal confirmed by MRI or CT within 6 months of randomization.
- •The patient's spleen volume is ≤ 10 x normal confirmed by MRI or CT within 6 months of randomization.
- •The patient has a serum creatinine < 2.0 mg/dL, an ASTand ALT < 2 x upper limit of normal and a total bilirubin < 2.0 x upper limit of normal.
- •Female patients of childbearing potential must have a negative pregnancy test within 2 weeks prior to randomization into the study.
排除标准
- •The patient is pregnant.
- •The patient has evidence of neurologic or pulmonary involvement with Gaucher disease confirmed by medical history.
- •The patient has evidence of current or prior bleeding varices or liver infarction requiring hospitalization confirmed by medical history.
- •The patient has evidence of pathologic bone fractures, medullary infarctions, lytic lesions or avascular necrosis secondary to Gaucher disease confirmed by skeletal evaluation within 6 months of randomization.
- •The patient has had a bone crisis (defined as pain with acute onset which requires immobilization of the affected area, narcotics for relief of pain and may be accompanied by periosteal elevation, increased white cell count, fever or debilitation of > 3 days) within 12 months of randomization.
- •Patient has received an investigational drug within 30 days of the start of their participation in this trial. Patients may not receive any other investigational product throughout the course of the study.
- •The patient has a clinically significant disease (with the exception of symptoms relating to Gaucher disease), including clinically significant cardiovascular, hepatic, immunologic, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstances that, in the opinion of the Investigator, would preclude participation in the trial or potentially decrease survival
- •Patient has a 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
Q2 Cerezyme
Patients receiving Cerezyme one infusion every 2 weeks (Q2).
干预措施: Cerezyme (Drug)
Q4 Cerezyme
Patients receiving Cerezyme one infusion every 4 weeks (Q4).
干预措施: Cerezyme (Drug)
结局指标
主要结局
Number of Participants With Clinical Success at Month 24/Discontinuation
时间窗: Month 24 (or at time of discontinuation)
Patients are considered to be a clinical success if ALL of the following are met: The patient's hemoglobin does not fall more than 1.25g/dL for women or 1.5 g/dL for men below the patient's baseline value, platelet count does not fall more than 25% below the patient's baseline value or does not fall below 80,000 mm3, liver and spleen volumes are not greater than 20% above the patient's baseline value, no evidence of bone disease progression, including no incidence of pathologic fractures, medullary infarctions, lytic lesions or avascular necrosis and has had no bone crises during the study.
次要结局
- Mean Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation.(Month 24 (or at time of discontinuation))
- Mean Composite Scores of the SF-36 Health Survey at Baseline(Baseline)
- Mean Change From Baseline in Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation(Baseline and Month 24 (or at time of discontinuation))