Sirolimus in Patients With Connective Tissue Disease Related Thrombocytopenia (CTD-TP): a Single-arm, Open-label Clinical Trial in China
试验速览
- 阶段
- 4 期
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Number of participants with complete remission of platelet count
研究概览
简要总结
We have designed a single-arm, open-label study of sirolimus in Chinese patients with refractory CTD-TP unresponsive to, or intolerant of, conventional medications at Peking Union Medical College Hospital (PUMCH). Patients received oral sirolimus at a starting dose of 2 mg per day for 3 days and a sequential dose of 1mg per day, with dose adjusted according to tolerance and to maintain a therapeutic range of 6-15 ng/mL. Patients were treated with sirolimus for more than 6 months. Safety outcomes and the efficacy endpoints were assessed in all patients who received at least one dose of treatment.
详细描述
Connective tissue disease related thrombocytopenia (CTD-TP) is one of the common visceral complications of connective tissue diseases (CTD) with a poor response to traditional therapy. To date, some successful cases were reported in sirolimus treating refractory CTD-TP as a newly therapeutic method. However, there is still a lack of prospective clinical trials. This study aims to evaluate the efficacy of sirolimus in the treatment of refractory CTD-TP. To investigate this issue, investigators have designed a single-arm, open-label clinical trial of refractory CTD-TP patients in Peking Union Medical College Hospital (PUMCH). In this study, the researchers will enroll 20 patients who have a poor response to at least 6 months' traditional treatment. The participants will receive oral sirolimus treatment for 12 months. The primary end point is the change of platelet count in CBC. Secondary end points include the change of CTD activity, the time to disease complete remission and all-cause serious adverse events (SAE).
Research Question:
Does sirolimus improve the prognosis in patients with connective tissue disease related thrombocytopenia (CTD-TP) in Chinese patients?
Specific aims:
- To determine the efficacy of sirolimus for the treatment of CTD-TP in Chinese patients
- To evaluate the safety of sirolimus for the treatment of CTD-TP in Chinese patients
- To analyze the clinical characteristics of CTD-TP in the sirolimus effective patients
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
- 16 Years 至 80 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with refractory CTD-TP unresponsive to, or intolerant of, conventional medications at Peking Union Medical College Hospital.
排除标准
- •new diagnosed CTD-IT patients
研究组 & 干预措施
Study Group
participants who received sirolimus treatment
干预措施: Sirolimus (Drug)
结局指标
主要结局
Number of participants with complete remission of platelet count
时间窗: 6 months
Complete remission of platelet count means the platelet count over 100X10\^9/L. every participants will take CBC as their screening times.
次要结局
- Number of participants with partial remission of platelet count(6 months)