NCT04656600已完成4 期
A Single Arm, Prospective, Open Label, Multicenter Study to Evaluate Efficacy and Safety of One-year Maximum Dosage in Chinese Label of Imiglucerase Treatment in Chinese Patients Who Are Diagnosed as Gaucher Disease Type Ⅲ
适应症
干预措施
相关药物
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- Sanofi
- 入组人数
- 12
- 试验地点
- 5
- 主要终点
- Adverse events
研究概览
简要总结
Primary Objective
- To evaluate the efficacy on hematologic manifestations of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ
- To evaluate the safety profile of imiglucerase in maximum dose in the label (60U/kg, IV biweekly) in Chinese patients.
Secondary Objective
- To evaluate the efficacy on viscera manifestations of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ
- To evaluate the efficacy on bone disease of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ
- To evaluate the effect on quality of life of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ
详细描述
Approximatively 14 months including a 12 months treatment period
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
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Cerezyme® / Imiglucerase
Experimental
Cerezyme® (imiglucerase for injection) is administered by intravenous infusion, 60 U/kg once every 2 weeks.
干预措施: Cerezyme® / Imiglucerase (Drug)
结局指标
主要结局
Adverse events
时间窗: Baseline to the end of 13 months
Number of participants with AEs
Changes in platelet count
时间窗: Baseline to the end of 12 months
The mean changes in platelet count
Changes in haemoglobin
时间窗: Baseline to the end of 12 months
The mean changes in haemoglobin
次要结局
- Changes in liver volume(Baseline to the end of 12 months)
- Changes in spleen volume(Baseline to the end of 12 months)
- Skeletal involvement(Baseline to the end of 12 months)
- Quality of life (QoL)(Baseline to the end of 3 months, 6 months, 9 months and 12 months)
研究者
研究点 (5)
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