A Multicenter Randomized Open-label Study of Chidamide Combined With High-dose Dexamethasone Versus High-dose Dexamethasone in the Management of Newly-Diagnosed Immune Thrombocytopenia
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 100
- 主要终点
- Sustained response
研究概览
简要总结
Recently, histone deacetylase inhibitors (HDACi) has been used for their anti-inflammatory and immunomodulatory activities. It has been shown that HDACi can alleviate graft-versus-host disease by enhancing the number and function of Foxp3+ Tregs. Our group found that low-dose HDACi alleviated thrombocytopenia in both passive and active murine models of ITP. Furthermore, low-dose HDACi attenuated macrophage phagocytosis of antibody-coated platelets, stimulated production of natural Foxp3+ Tregs, promoted peripheral conversion of T cells into Tregs, and restored Treg suppressive function in vivo and in vitro. The project was undertaking by Qilu Hospital of Shandong University and other 10 well-known hospitals in China. In order to report the efficacy and safety of the low dose chidamide combined with high-dose dexamethasone versus high-dose dexamethasone in the management of ITP.
详细描述
Primary immune thrombocytopenia (ITP) is an autoimmune bleeding disorder with low platelet count. Low-dose HDACi alleviated thrombocytopenia in both passive and active murine models of ITP. In this multicentre, open-label, randomized controlled trial, newly diagnosed ITP patients will be enrolled from 11 tertiary medical centres in China. Participants will be randomly assigned into the combination group or the monotherapy group by masked statisticians in a 1:1 ratio. The primary endpoints are sustained response at month 6. The secondary outcomes include initial response, time to response, duration of response, bleeding score, health-related quality of life assessment, and safety issue. This study will compare the efficacy and safety of low dose chidamide combined with high-dose dexamethasone versus high-dose dexamethasone monotherapy in adults with primary immune thrombocytopenia.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 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
- •newly diagnosed ITP patients need of treatment(s) to minimize the risk of clinically significant bleeding primary ITP confirmed by excluding other supervened causes of thrombocytopenia
排除标准
- •pregnancy hypertension cardiovascular disease diabetes liver and kidney function impairment HCV, HIV, HBsAg seropositive status patients with systemic lupus erythematosus and/or antiphospholipid syndrome
研究组 & 干预措施
Chidamide plus HD-DXM
Chidamide (orally at 5 mg biw for 24 weeks)
HD-DXM (orally at 40 mg daily for 4 days)
干预措施: Chidamide (Drug)
Chidamide plus HD-DXM
Chidamide (orally at 5 mg biw for 24 weeks)
HD-DXM (orally at 40 mg daily for 4 days)
干预措施: HD-DXM (Drug)
HD-DXM
HD-DXM (orally at 40 mg daily for 4 days )
干预措施: HD-DXM (Drug)
结局指标
主要结局
Sustained response
时间窗: 6 month
The maintenance of platelet count ≥ 30 x 10\^9/L, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 6-month follow-up.
次要结局
- Number of patients with bleeding(6 month)
- Number of patients with adverse events(6 month)
- Initial response(day 14)
- Time to response(6 month)
- Duration of response (DOR)(6 month)
- Health-related quality of life assessment(6 month)
研究者
Ming Hou
Professor and Director
Shandong University