A Prospective, Open-label, Multicenter，Single-armed Study of Orelabrutinib in the Treatment of Refractory Primary Immune Thrombocytopenia (ITP)
试验速览
- 阶段
- 2 期
- 发起方
- 入组人数
- 40
- 主要终点
- Initial overall response to Orelabrutinib
研究概览
简要总结
BTK participates in a variety of signal transduction of innate and adaptive immunity, and has an important role in cell proliferation, differentiation and apoptosis. The impact of BTK inhibitors on hematological malignancies and autoimmune diseases has been well studied. This project was undertaking by Qilu Hospital of Shandong University in China. In order to report the efficacy and safety of the selective BTK inhibitor Orelabrutinib in the management of refractory ITP.
详细描述
The investigators are undertaking a prospective, open-lable, multicentre trial of 40 refractory ITP adult patients in China. Eligible participants will receive Orelabrutinib in 50 mg po. qd, every 4 weeks for one cycle and it will be given 3 cycles. For non-responders who were well tolerated at 12 weeks of follow-up, the treatment could be extended to 6 cycles. The treatment will be discontinued after 6 months without blood index reaction. In order to report the efficacy and safety of Orelabrutinib in the management of refractory ITP, platelet count, bleeding and other symptoms will be evaluated before and after treatments. Adverse events are also recorded throughout the study.
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 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Meet the diagnostic criteria for primary immune thrombocytopenia
- •To show a platelet count < 30 * 10^9/L, or with bleeding manifestations, or both
- •Willing and able to sign written informed consent
- •Meet the diagnostic criteria of refractory ITP according to Chinese guidelines
排除标准
- •Secondary thrombocytopenia
- •severe immune-deficiency or history of primary immunodeficiency
- •active or previous malignancy
- •HIV virus infection, tuberculosis, or other active infection (sepsis, pneumonia, or abscess)
- •pregnancy or lactation
- •hypertension
- •cardiovascular diseases
- •severe liver or kidney function impairment
- •psychosis
- •osteoporosis
- •inflammatory bowel disease or gastric disease
- •arterial or venous thromboembolism within the 6 months before screening or patients who required anticoagulant treatment
- •an organ or haematopoietic stem-cell transplantation
- •neutrophil count of less than 1500 cells per mm³; glycosylated haemoglobin less than 8%; partial thromboplastin time 1∙5 times or less the upper limit of normal (ULN)
- •clinical electrocardiogram changes
- •neoplastic disease within the past 5 years
- •corrected QT interval greater than 450 ms for men and greater than 470 ms for women;
- •substance misuse within the previous 12 months; and those who could not adhere to the protocol or were planning to have a surgical procedure in 6 months.
研究组 & 干预措施
Orelabrutinib
Orelabrutinib 50mg po qd
干预措施: Orelabrutinib (Drug)
结局指标
主要结局
Initial overall response to Orelabrutinib
时间窗: 14 days after the first dose of Orelabrutinib
Complete response was defined as a platelet count of 100×10⁹ cells per L or higher and an absence of bleeding. Partial response was defined as a platelet count of 30×10⁹ cells per L or higher, but less than 100×10⁹ cells per L, and at least a doubling of the baseline platelet count and an absence of bleeding. Platelet counts were confirmed on two separate occasions at least 7 days apart when defining complete response or partial response. No response was defined as a platelet count of less than 30×10⁹ cells per L, or less than two-times increase from baseline platelet count, or bleeding.
Sustained overall response to Orelabrutinib
时间窗: A response lasting for at least 6 months without any additional intervention specific to primary immune thrombocytopenia was defined as a sustained response
Complete response was defined as a platelet count of 100×10⁹ cells per L or higher and an absence of bleeding. Partial response was defined as a platelet count of 30×10⁹ cells per L or higher, but less than 100×10⁹ cells per L, and at least a doubling of the baseline platelet count and an absence of bleeding. Platelet counts were confirmed on two separate occasions at least 7 days apart when defining complete response or partial response. No response was defined as a platelet count of less than 30×10⁹ cells per L, or less than two-times increase from baseline platelet count, or bleeding.
次要结局
- Time to response(An average of 6 months)
- Duration of response(through study completion, an average of 1 year)
- Therapy associated adverse events(Up to 1 year)
研究者
Ming Hou
Professor and Director
Shandong University