Thrombopoietin-receptor Agonist-immunomodulation in Young Adult Primary Immune Thrombocytopenia (ITP): A Multi-center Open Label Trial With Romiplostim
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 15
- 试验地点
- 5
- 主要终点
- Change in Interleukin (IL)-4 concentrations (pg/ml) from baseline to week 22
研究概览
简要总结
The study aims to investigate immunomodulatory effects of thrombopoietin-receptor Agonist (TPO-RA) in patients with primary ITP, who failed first-line therapy or who became intolerant to it. It is hypothesized that the early phase of this autoimmune disease may exhibit a stronger immunomodulatory potential in response to a stimulus, such as romiplostim. Such a process may subsequently be capable to induce regulatory mechanisms or tolerance.
Romiplostim (a thrombopoietin-receptor agonist, TPO-RA) will be administered subcutaneously once weekly over 22 weeks with a starting dose of 1mcg/kg body weight. The dose will be adjusted based on platelet counts as described in the summary of Product Characteristics (SmPC).
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 45 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Informed consent as documented by signature (see informed consent form)
- •Primary ITP according to the definition of Rodeghiero et al. (52) and a platelet count of <30x109/l
- •Age range: 18-45 years
- •Previously treated patients, with failure or intolerance to first-line therapy, or relapse after first-line therapy, i.e. corticosteroids, intravenous immunoglobulin (IVIG), or anti-D immunoglobulins
排除标准
- •Adults older than 45 and children younger than 18 years
- •Platelet count higher than 30x109/l at time of screening
- •Suspicion of secondary ITP
- •Positive family history for ITP
- •Presence or history of autoimmune disease as judged by the investigator
- •Hepatosplenomegaly
- •Presence or history of relevant hepatic disease as judged by the investigator
- •Presence or history of thromboembolic disease as judged by the investigator
- •Patients with splenectomy
- •Women who are pregnant or breast feeding
- •Intention to become pregnant during the course of the study
- •Lack of safe double contraception (see 7.1)
- •Any vaccination 2 weeks prior start of the study
- •Drugs with a known impact on the immune system or on platelet function must be recorded and an exclusion of the study should be discussed with the study center
- •Known or suspected non-compliance, drug or alcohol abuse
- •Inability to follow the procedures of the study, e.g. due to language problems, psychological disorders, dementia of the study subject
- •Participation in another study with investigational drug within the 30 days preceding and during the present study
- •Previous enrolment into the current study
- •Previous treatment with romiplostim or eltrombopag
- •Hypersensitivity to the active substance or to any of the excipients or to E. coli derived proteins
- •Enrolment of the investigator, his/her family members, employees and other dependent persons
研究组 & 干预措施
Romiplostim
Romiplostim (a thrombopoietin-receptor agonist, TPO-RA) will be administered subcutaneously once weekly over 22 weeks with a starting dose of 1mcg/kg body weight. The dose will be adjusted based on platelet counts as described in the summary of Product Characteristics (SmPC). Followup examination at week 52.
干预措施: romiplostim (Drug)
结局指标
主要结局
Change in Interleukin (IL)-4 concentrations (pg/ml) from baseline to week 22
时间窗: baseline and 22 weeks
The primary aim of the study is to demonstrate an immunomodulatory effect of the study drug. Investigators expect a shift in the Th1/Th2 balance towards Th2. The primary outcome is to compare the pre- and post-treatment IL-4 concentrations (pg/ml) of all included patients (Th2 profile). Assessment of change in pre- and post-treatment IL-4 concentrations (pg/ml).
次要结局
- Change in immunomodulation as assessed by mRNA of cytokines between baseline and week 10(baseline and 10 weeks)
- Change in immunomodulation as assessed by immune cell characteristics between baseline and week 22(baseline and 22 weeks)
- Change in immunomodulation as assessed by cytokine concentrations between baseline and week 10(baseline and 10 weeks)
- Change in immunomodulation as assessed by cytokine concentrations between baseline and week 52(baseline and 52 weeks)
- Clinical response between baseline and week 52: frequency of use of rescue treatment(baseline and week 52)
- Change in immunomodulation as assessed by immune cell characteristics between baseline and week 10(baseline and 10 weeks)
- Change in immunomodulation as assessed by messenger ribonucleic acid (mRNA) of cytokines between baseline and week 22(baseline and 22 weeks)
- Change in immunomodulation as assessed by mRNA of immune cells between baseline and week 22(baseline and 22 weeks)
- Change in immunomodulation as assessed by mRNA of immune cells between baseline and week 10(baseline and 10 weeks)
- Change in immunomodulation as assessed by cytokine concentrations between baseline and week 22(baseline and 22 weeks)
- Clinical response between baseline and week 52: number of days in hospital(baseline and 52 weeks)
- Change in immunomodulation as assessed by immune cell characteristics between baseline and week 52(baseline and 52 weeks)
- Change of immunomodulation as assessed by mRNA of cytokines between baseline and week 52(baseline and 52 weeks)
- Clinical response between baseline and week 52: number of severe bleeding(baseline and 52 weeks)
- Clinical response between baseline and week 52: platelet more than >100G/l(baseline and 52 weeks)
- Change in immunomodulation as assessed by mRNA of immune cells between baseline and week 52(baseline and 52 weeks)