A Prospective, Multicenter Clinical Trial of Chidamide in the Management of Refractory Immune Thrombocytopenia
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 61
- 试验地点
- 1
- 主要终点
- Overall response at week 12
研究概览
简要总结
Primary immune thrombocytopenia (ITP) is an autoimmune bleeding disorder. Increased macrophage phagocytosis of antibody-coated platelet as well as decreased number and/or impaired function of CD4+CD25+Foxp3+ regulatory T (Treg) cells have been shown to participate in the pathogenesis of ITP. Our preclinical data revealed that chidamide, a histone deacetylase inhibitor (HDACi), could attenuate macrophage phagocytosis of antibody-coated platelets, stimulate production of natural Foxp3+ Tregs, and upregulate CTLA4 expression through modulation of histone H3K27 acetylation. The project was undertaking by Qilu Hospital of Shandong University in China. In order to evaluate the efficacy and safety of chidamide at two different dosage regimens in adult patients with refractory ITP.
详细描述
In this prospective, open-label, multicenter, randomized clinical trial, refractory ITP adult patients will be enrolled from five medical centers in China. Eligible participants will be randomly assigned 1:1 to receive chidamide at either 2.5 or 5 mg twice per week. The primary endpoint is the overall response at week 12. Complete response was defined as a platelet count at or above 100×10^9/L and an absence of bleeding. Partial response was defined as a platelet count at or above 30×10^9/L but less than 100×10^9/L and at least a doubling of the baseline platelet count and an absence of bleeding. No response was defined as a platelet count of less than 30×10^9 cells per L, or less than two-times increase from baseline platelet count, or bleeding. The secondary enpoints included 6-month sustained response, time to response (TTR), duration of response, bleeding scores, health-related quality of life assessment and adverse events (AEs). This study will compare the efficacy and safety of chidamide in two different dosage regimens in adult patients with refractory.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 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
- •Participant must be at least 18 years of age at the time of the screening.
- •Participant may be male or female.
- •Participant has a confirmed diagnosis of ITP according to the 2019 International Working Group assessment for more than 6 months at screening.
- •Participant who didn't respond or relapsed after previous first-line treatment, and lack of response to rituximab, TPO agents, or splenectomy.
- •Bone marrow biopsy is performed in participants over 60 years to exclude hematological malignancies.
排除标准
- •Participant has evidence of a secondary cause of immune thrombocytopenia or to drug treatments or participant has a multiple immune cytopenia, e.g. Evan's syndrome.
- •Participant with the following conditions:severe dysfunction of the heart, kidney, liver, or lung; severe immunodeficiency; malignancy; HIV; hepatitis B or C virus infection; pregnancy or lactation.
研究组 & 干预措施
A
Chidamide 2.5 mg po twice per week
干预措施: Chidamide (Drug)
B
Chidamide 5 mg po twice per week
干预措施: Chidamide (Drug)
结局指标
主要结局
Overall response at week 12
时间窗: week 12
The primary endpoint was the overall response at week 12.Complete response was defined as a platelet count at or above 100×10\^9/L and an absence of bleeding. Partial response was defined as a platelet count at or above 30×109/L but less than 100×10\^9/L and at least a doubling of the baseline platelet count and an absence of bleeding. No response was defined as a platelet count of less than 30×10\^9 cells per L, or less than two-times increase from baseline platelet count, or bleeding.
次要结局
- Sustained response(6 months)
- Duration of response(12 months)
- Bleeding scores(12 months)
- Therapy associated adverse events(up to 1 year per subject)
- Health-related quality of life assessment(12 weeks)
- Time to response(12 weeks)
研究者
Ming Hou
Professor and Director
Shandong University