Special Drug Use Surveillance of ADZYNMA Intravenous 1500 (All-Case Investigation)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Number of Participants who Experience at Least One Treatment-Emergent Adverse Events (TEAE)
研究概览
简要总结
This study is a survey in Japan of recombinant ADAMTS13 used to treat or to prevent participants with congenital thrombotic thrombocytopenic purpura (cTTP). The study sponsor will not be involved in how the participants are treated but will provide instructions on how the clinics will record what happens during the study.
The main aim of the study is to check for side effects related from recombinant ADAMTS13 and to check if recombinant ADAMTS13 improves or prevents cTTP.
During the study, participants with cTTP will take recombinant ADAMTS13 intravenous injection according to their clinic's standard practice. The study doctors will check for side effects from recombinant ADAMTS13 for 18 months.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •- All participants with congenital thrombotic thrombocytopenic purpura (cTTP), treated with recombinant ADAMTS13
排除标准
- 未提供
研究组 & 干预措施
Recombinant ADAMTS13
Participants will receive recombinant ADAMTS13 intravenous injection.
干预措施: Recombinant ADAMTS13 (Drug)
结局指标
主要结局
Number of Participants who Experience at Least One Treatment-Emergent Adverse Events (TEAE)
时间窗: Up to 18 Months
次要结局
- Percent Change in Observed Platelet Count from Baseline at the End of Treatment(Up to 18 Months)
- Percent Change in Observed ADAMTS13 Activity from Baseline at the End of Treatment(Up to 18 Months)
- Percent Change in Observed ADAMTS13 Inhibitor from Baseline at the End of Treatment(Up to 18 Months)
- Number of Participants with Thrombotic Thrombocytopenic Purpura (TTP) Events on Periodic Replacement Therapy(Up to 18 Months)