Safety and Efficacy of CD123-Targeted CAR-T 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
- 40
- 试验地点
- 1
- 主要终点
- Adverse events that related to treatment
研究概览
简要总结
This is a single arm study to evaluate the efficacy and safety of CD123-targeted CAR-T cells therapy for patients with relapsed/refractory Acute Myeloid Leukemia.
详细描述
There are limited options for treatment of relapse/refractory Acute Myeloid Leukemia. CD123 is expressed on most myeloid leukemia cells so it is an 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 relapsed/refractory Acute Myeloid 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
- 3 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 acute myeloid leukemia, and meet one of the following conditions:
- •Failed to standard chemotherapy regimens;
- •Relapse after complete remission, high-risk and / or refractory patients ;
- •Relapse after hematopoietic stem cell transplantation;
- •Evidence for cell membrane CD123 expression;
- •All genders, ages: 3 to 75 years；
- •The expect time of survive is above 12 weeks;
- •No serious mental disorders ;
- •Left ventricular ejection fraction ≥50%
- •Sufficient hepatic function defined by ALT/AST≤3 x ULN and bilirubin≤2 x ULN;
- •Sufficient renal function defined by creatinine clearance≤2 x ULN;
- •Sufficient pulmonary function defined by indoor oxygen saturation≥92%;
- •With single or venous blood collection standards, and no other cell collection contraindications;
- •Ability and willingness to adhere to the study visit schedule and all protocol requirements.
排除标准
- •Have received CAR-T therapy or other genetically modified cell therapy before screening；
- •Participated in other clinical research within 1 month before screening；
- •Have received the following anti-tumor treatment before screening: Have received chemotherapy, targeted therapy or other experimental drug treatment within 4 weeks, except those who have confirmed disease progression after treatment;
- •Live attenuated vaccine within 4 weeks before screening;
- •Convulsion or stoke within past 6 months;
- •Previous history of other malignancy;
- •Presence of uncontrolled active infection;
- •Subjects with positive HBsAg or HBcAb positive and peripheral blood HBV DNA titer is higher than the lower limit of detection of the research institution; HCV antibody positive and peripheral blood HCV RNA titer is higher than the lower limit of detection of the research institution; HIV antibody positive; syphilis primary screening antibody positive;
- •Pregnant or breasting-feeding women;
- •Any situation that investigators regard not suitable for attending in this study or may affect the data analysis.
结局指标
主要结局
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)
The response rate of CD123 CAR-T treatment in patients with relapse/refractory Acute Myeloid Leukemia that treatment by CD123 CAR-T cells therapy
时间窗: 6 months
The response rate of CD123 CAR-T treatment will be recorded and assessed according to the National Comprehensive Cancer Network Guideline
次要结局
- Rate of CD123 CAR-T cells in bone marrow and peripheral blood(2 years)
- Cellular kinetics of CD123 positive cells in bone marrow(1 years)
- Levels of cytokines in serum(3 months)
- Duration of Response (DOR) of CD123 CAR-T treatment in patients with refractory/relapsed acute myeloid leukemia(2 years)
- Quantity of CD123 CAR copies in bone marrow and peripheral blood(2 years)
- Progress-free survival(PFS) of CD123 CAR-T treatment in patients with refractory/relapsed acute myeloid leukemia(2 year)
- Overall survival(OS) of CD123 CAR-T treatment in patients with refractory/relapsed acute myeloid leukemia(2 years)