Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of Open Label Study With Multiple (and for Non Responders) Escalating Subcutaneous Doses of BI 655064 Once a Week in Patients With Chronic Primary Immune Thrombocytopenic Purpura.
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 6
- 试验地点
- 2
- 主要终点
- Number of Patients With Response in Platelet Count
研究概览
简要总结
BI 655064 will be administered subcutaneously once weekly in patients with immune thrombocytopenic purpura (ITP) for up to 12 weeks.
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
BI 655064 120 mg / 180 mg
The patients were administered 120 mg BI 655064 solution for subcutaneous injection q1w (once a week) subcutaneously for 4 weeks.
Patients who showed an increase in platelet count above or equal to 100 x 10^9/L continued treatment with 120 mg BI 655064 solution for subcutaneous injection q1w for additional 8 weeks, followed by 12 weeks of follow-up.
Patients whose platelet count stayed below 100 x 10^9/L continued treatment for 2 weeks with 180 mg BI 655064 solution for subcutaneous injection q1w followed by 120 mg BI 655064 solution for subcutaneous injection q1w for additional 6 weeks, followed by 12 weeks of follow-up.
干预措施: BI 655064 (Drug)
结局指标
主要结局
Number of Patients With Response in Platelet Count
时间窗: Up to 12 weeks.
This outcome measure presents number of patients with a response in platelet count. Response was defined as: \[A\] An increase in platelet count by greater than 20 x 10\^9/L from baseline at any time-point between Week 1 and Week 12, and \[B\] Platelet count above 50 x 10\^9/L at any time point between Week 1 and Week 12 with no rescue therapy. Baseline was defined as the platelet count at Visit 2 before administration of BI 655064. In case the patient fulfilled only one of the conditions, he/she was considered a non-responder.
次要结局
- Number of Subjects With Drug-related Adverse Events(From first drug administration until end of the Residual Effect Period (REP), up to 6 weeks.)
- Number of Patients Reaching the Cut-off Point for Immune Thrombocytopenic Purpura [ITP](Up to 12 weeks.)