Multi-center Phase II Prospective Study for the Treatment of Chidamide and Venetoclax in Patients With All-trans Retinoic Acid (ATRA) and Arsenic (As) Resistant Acute Promyelocytic Leukemia (APL)
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 30
- 试验地点
- 5
- 主要终点
- complete remission and complete remission with incomplete recovery of peripheral blood count
研究概览
简要总结
Based on the current treatment with retinoic acid (ATRA) and arsenic (As), most patients with APL achieved long-term survival. There are few patients relapsed and became refractory to the RA and As treatment. In our pre-clinical study, we found that targeting histone deacetylase inhibitor 3 (HDAC3)degraded PML-RARa oncoprotein and induced differentiation and apoptosis of RA and As resistant APL in vitro and in vivo. In this study, we evaluate the efficacy and feasibility of combination therapy for HDAC3 inhibitor and venetoclax in patients with refractory APL.
详细描述
For patients with newly-diagnosed acute promyelocytic leukemia (APL), the combination therapy retinoic acid (ATRA) combined with arsenic (As) is the mainstay upfront treatment. Most patients enjoy long-term survival with or without chemotherapy. Even though with such good prognosis, there are still some patients relapsed and eventually became refractory to RA and As treatment. In our pre-clinical study, we demonstrate that targeting histone deacetylase inhibitor 3 (HDAC3) could degrade PML-RARa oncoprotein and induce differentiation and apoptosis of RA and As resistant APL in vitro and in vivo. In this multi-center prospective study, we evaluate the efficacy and feasibility of combination therapy for HDAC3 inhibitor (chidamide) and venetoclax in patients with refractory APL.
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 70 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with PML-RARα+ APL
- •Patients in non-remission status after treatment of RA combined with As
- •Patients with life expectance >=3 months
- •Inform consent provided
排除标准
- •Patients with incontrollable infection
- •Patients with life-expectancy less than 2 months
- •Patients with abnormal liver (>3XN) and renal function (>3XN）
研究组 & 干预措施
Chi-Ven treatment
Patients receive chidamide and venetoclax treatment
干预措施: Chidamide+venetoclax (Drug)
结局指标
主要结局
complete remission and complete remission with incomplete recovery of peripheral blood count
时间窗: Day 42
patients with \<5% promyelocytes in bone marrow with or without full recovery of peripheral cell count
次要结局
- Non-relapse mortality(1 year)
- Early death(42 days)
- Overall survival(1 year)
- Leukemia-free survival(1 year)
- relapse(1 year)
研究者
Jiong HU
Head, Blood & Marrow Transplantation Center, Rui Jin Hospital
Shanghai Jiao Tong University School of Medicine