CD123-Targeted CAR-T Cell Therapy for Relapsed/Refractory Acute Myeloid Leukemia
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 45
- 试验地点
- 1
- 主要终点
- The response rate of CD123 CAR-T treatment in patients with relapse/refractory AML that treatment by CD123 CAR-T cells therapy
研究概览
简要总结
There are limited options for treatment of relapse/refractory acute myeloid leukemia (AML). CD123 CAR-T cells may have an attractive and permanent effect on anti-tumor. This study purpose to estimate the safety and efficiency of CD123 CAR-T cells to patients with relapse/refractory AML.
详细描述
CD123 is expressed on most myeloid leukemia cells so it is a ideal target for CAR-T. Some researches have revealed that CD123 is a marker of leukemia stem cells, which indicates that the eradication of CD123 cells may prevent relapse of leukemia. In this study, investigators will evaluate the safety and efficacy of CAR-T targeting CD123 in patients with Acute Myelocytic Leukemia. The primary goal is safety and efficiency assessment, including adverse events and disease status after treatment.
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
- 2 Years 至 75 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed written informed consent；
- •Diagnose as Relapsed/Refractory AML, and meet one of the following conditions：
- •With persistent disease after at least two lines of therapy；
- •Relapse to the last line of therapy in 6 months，as known as early recurrence;
- •Relapse to the last line of therapy after 6 months, but refractory to this last line of therapy；
- •Relapse more than once. The definition of relapse: Reappearance of blasts in the blood or bone marrow (>5%) or in any extramedullary site after a CR (the most common are CNS and testicular leukemia).
- •Evidence for cell membrane CD123 expression;
- •The expect time of survive is above 3 months;
- •Ages: 2 to 75 years;
- •All genders;
- •The patients that diagnosis as high risks, relapse/refractory or inconformity criteria to other therapy；
- •No serious mental disorders;
- •Left ventricular ejection fraction ≥40%;
- •Sufficient hepatic function defined by ALT/AST<5 x ULN and bilirubin≤34.2μmol/L;
- •Sufficient renal function defined by creatinine clearance <220μmol/L;
- •Sufficient pulmonary function defined by indoor oxygen saturation≥92%;
- •No other illness may conflict with the protocol (e.g. autoimmune diseases, immune deficiency and organ transplantation；
- •Ability and willingness to adhere to the study visit schedule and all protocol requirements.
排除标准
- •Previous history of other malignancy;
- •Presence of uncontrolled active infection;
- •Evidence of disorder that need the treatment by glucocorticoids;
- •Active or chronic GVHD；
- •The patients treatment by inhibitor of T cell；
- •Pregnant or breasting-feeding women;
- •Any situation that investigators regard not suitable for attending in this study (e.g. HIV , HCVinfection or intravenous drug addiction) or may affect the data analysis.
结局指标
主要结局
The response rate of CD123 CAR-T treatment in patients with relapse/refractory AML that treatment by CD123 CAR-T cells therapy
时间窗: 2 years
The response rate of CD123 CAR-T treatment will be recorded and assessed according to the National Comprehensive Cancer Network Guideline.
Adverse events that related to treatment
时间窗: 2 years
Therapy-related adverse events will be recorded and assessed according to the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE, Version 5.0).
次要结局
- Overall survival(OS) of CD123 CAR-T treatment in patients with refractory/relapsed AML(2 years)
- Cellular kinetics of CD123 CAR-T in Bone marrow(2 years)
- Progress-free survival(PFS) of CD123 CAR-T treatment in patients with refractory/relapsed AML(2 years)
- Duration of Response (DOR) of CD123 CAR-T treatment in patients with refractory/relapsed AML(2 years)
- Cellular kinetics of CD123 CAR-T in Blood(2 years)
- Cellular kinetics of CD123 positive cells in Bone marrow(1 years)