An Open-label Multicenter Trial to Study the Efficacy and Safety of Caplacizumab in Japanese Patients With Acquired Thrombotic Thrombocytopenic Purpura
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Sanofi
- 入组人数
- 21
- 试验地点
- 12
- 主要终点
- Proportion of participants with a recurrence of acquired thrombotic thrombocytopenic purpura (aTTP)
研究概览
简要总结
Primary Objective:
To evaluate the effect of caplacizumab on prevention of recurrence of aTTP (proportion of participants with a recurrence of aTTP) during the overall study period.
Secondary Objectives:
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To evaluate effect of caplacizumab on
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prevention of recurrence of TTP (the number of recurrences of TTP) during overall study period.
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a composite endpoint consisting of aTTP-related mortality, recurrence of aTTP and major thromboembolic events during study drug treatment
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restoring platelet counts as a measure of prevention of further microvascular thrombosis
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refractory disease
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biomarkers of organ damage: LDH, cardiac troponin I, serum creatinine
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plasma exchange (PE) parameters (days of PE and volume of plasma used), days in intensive care unit, days in hospital
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cognitive status of Japanese patients
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To evaluate safety profile of caplacizumab in Japanese patients
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To evaluate effect of caplacizumab on pharmacodynamic (PD) markers in Japanese patients
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To evaluate pharmacokinetic (PK) profile of caplacizumab in Japanese patients
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To evaluate immunogenicity of caplacizumab in Japanese patients
详细描述
Study duration per participant is approximately 2 months up to approximately 6 months in case of treatment extension and recurrence during the study drug 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
- 18 Years 至 —（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
Caplacizumab
Eligible study participants will receive caplacizumab in addition to standard of care such as daily plasma exchange (PE) and corticosteroid treatment (mandatory), immunosuppressive treatment (if needed)
干预措施: Caplacizumab (ALX-0081) (Drug)
Caplacizumab
Eligible study participants will receive caplacizumab in addition to standard of care such as daily plasma exchange (PE) and corticosteroid treatment (mandatory), immunosuppressive treatment (if needed)
干预措施: Plasma exchange (PE) (Drug)
Caplacizumab
Eligible study participants will receive caplacizumab in addition to standard of care such as daily plasma exchange (PE) and corticosteroid treatment (mandatory), immunosuppressive treatment (if needed)
干预措施: Corticosteroid treatment (Methylprednisolone or prednisolone) (Drug)
Caplacizumab
Eligible study participants will receive caplacizumab in addition to standard of care such as daily plasma exchange (PE) and corticosteroid treatment (mandatory), immunosuppressive treatment (if needed)
干预措施: Immunosuppressive treatment (eg, rituximab) (Drug)
结局指标
主要结局
Proportion of participants with a recurrence of acquired thrombotic thrombocytopenic purpura (aTTP)
时间窗: Approximately 2 months up to approximately 6 months
Proportion of participants with a recurrence of aTTP during the overall study period. The success criterion for this study is proportion of evaluable participants (per-protocol population) with a recurrence of aTTP during the overall study period to be 20% or less.
次要结局
- Proportion of participant who have a platelet count ≥150,000/μL(Approximately 2 months up to approximately 6 months)
- Number of recurrences of TTP(Approximately 2 months up to approximately 6 months)
- Proportion of participants with composite endpoint consisting of aTTP-related mortality, recurrence of aTTP and major thromboembolic events(Approximately 2 months up to approximately 6 months)
- Number of days to plasma exchange(Approximately 2 months up to approximately 6 months)
- Time to platelet count response(Approximately 2 months up to approximately 6 months)
- Time to stop of daily plasma exchnage (PE)(Approximately 2 months up to approximately 6 months)
- Change from baseline in the standardized mini mental state exam (SMMSE) total score(Approximately 2 months up to approximately 6 months)
- Pharmacodynamic (PD) markers(Approximately 2 months up to approximately 6 months)
- Immunogenicity of caplacizumab(Approximately 2 months up to approximately 6 months)
- Proportion of participants with refractory TTP(Approximately 2 months up to approximately 6 months)
- Pharmacokineticks: plasma concentration(Approximately 2 months up to approximately 6 months)
- Time to normalization of 3 organ damage marker levels(Approximately 2 months up to approximately 6 months)
- Total volume of plasma(Approximately 2 months up to approximately 6 months)
- Number of days in ICU and in hospital(Approximately 2 months up to approximately 6 months)
- Proportion of participants with at least one treatment emergent thromboembolic event(Approximately 2 months up to approximately 6 months)
- Number of patients with treatment emergent adverse events(Approximately 2 months up to approximately 6 months)