A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase in Patients With Gaucher Disease Treated With Imiglucerase
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 31
- 试验地点
- 10
- 主要终点
- Hemoglobin
研究概览
简要总结
This is a multi-center, open-label, switchover trial to assess the safety of taliglucerase alfa in 30 patients with Gaucher disease who are currently being treated with imiglucerase (Cerezyme®) enzyme replacement therapy.
详细描述
This is a multi-center, open-label, switchover trial to assess the safety of taliglucerase alfa in 30 patients with Gaucher disease who are currently being treated with imiglucerase (Cerezyme®) ERT. Eligible patients will enter a 12-week Stability Evaluation Period to establish the stability of their disease. Patients with stable disease will then be switched from their imiglucerase treatment to receive intravenous (IV) infusions of taliglucerase alfa every two weeks for a total of 20 IV infusions. The dose of taliglucerase alfa will be equal to each patient's previous imiglucerase dose. The infusions will be administered at the selected investigational site (clinic/hospital), infusion center, or at home. 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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males and females, 2 years or older
- •Confirmed diagnosis of Gaucher disease by the enzymatic activity assay
- •Stable Gaucher disease
- •Treatment with imiglucerase (Cerezyme®) for at least 2 years and on a stable maintenance regimen (dose and regimen unchanged, except for situation of drug shortage) for at least the last six months
- •Able to provide written informed consent
排除标准
- •Currently taking another experimental drug for any condition
- •History of allergy to carrots
- •History of allergy to beta lactam antibiotics
- •Previous infusion reaction suspected to be allergic in nature to Cerezyme® or Ceredase® or receiving premedication to prevent infusion reactions
- •Presence of HIV and/or HBsAg and/or hepatitis C infection
- •Presence of unresolved anemia due to iron, folic acid or vitamin B12 deficiency
- •Presence of any significant comorbidity that could confound the interpretation of the clinical response to taliglucerase alfa
- •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
Taliglucerase alfa
Open label taliglucerase alfa treatment
干预措施: Taliglucerase alfa (Drug)
结局指标
主要结局
Hemoglobin
时间窗: Every 3 months from Baseline to Month 9
次要结局
未报告次要终点