Efficacy of Concomitant Use of Azacitidine and Homoharringtonine in Children With Juvenile Myelomonocytic Leukemia
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Juvenile Myelomonocytic Leukemia (JMML) response rate at end of 4 Months
研究概览
简要总结
Juvenile myelomonocytic leukemia (JMML) has a poor prognosis in general, with hematopoietic stem cell transplant (HSCT) remaining the standard of care for cure. Azacitidine and homoharringtonine, which exhibited a anticancer mechanism and has been widely used in patients with relapse/refractory acute myeloid leukemia (AML). However, the efficacy of these agents has not been elucidated in children with JMML. The primary objective is to assess the treatment effect on response rate in this subject population.
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
- 1 Month 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Newly diagnosed JMML, with PB and BM confirming diagnosis prior to informed consent signature, with one of the following:
- •somatic mutation in PTPN11
- •somatic mutation in KRAS
- •somatic mutation in NRAS and HbF % > 5x normal value for age
- •clinical diagnosis of neurofibromatosis Type 1.
排除标准
- •Any condition that would prevent the subject from participating in the study.
- •Any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if participated in the study.
- •Any condition that confounds the ability to interpret data from the study.
研究组 & 干预措施
Intervention group
干预措施: Azacitidine and homoharringtonine (Drug)
结局指标
主要结局
Juvenile Myelomonocytic Leukemia (JMML) response rate at end of 4 Months
时间窗: Up to 4 Months
Defined as proportion of patients with sustained clinical complete remission \[cCR\] or clinical partial remission \[cPR\] according to the International JMML response criteria in Niemeyer 2014 at 3 months (28 days cycles). Response must be sustained for at least 4 weeks either in the 4-week period preceding or succeeding 3 months (ie, sustained over the period minimum 2 months to end of 3 months, or end of 3 months to end of 4 months).
次要结局
未报告次要终点
研究者
Lipeng Liu
Dr
Air Force Military Medical University, China