An Open-label, Single-arm Clinical Study of EX02 CAR-T Therapy for Relapsed and Refractory Acute Myeloid Leukemia
试验速览
- 阶段
- 早期 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 9
- 试验地点
- 1
- 主要终点
- Treatment-Emergent Adverse Events (TEAE)
研究概览
简要总结
This is a early Phase 1 open-label, single-arm clinical study of EX02 CAR-T therapy for relapsed and refractory acute myeloid leukemia.
Each participant will undergo leukapheresis after enrolment, receive treatment of the conditioning chemotherapy of cyclophosphamide and fludarabine, and an an intravenous infusion of CAR-T cells.
Each participant will proceed through the following study procedures:
- Screening
- Enrollment/Leukapheresis
- Conditioning chemotherapy
- CAR T treatment
- Post-treatment assessment
- Long-term follow-up
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
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •According to the Chinese Adult Acute Myeloid Leukemia (AML) Diagnosis and Treatment Guidelines (2023 edition/previous edition), patients diagnosed with acute myeloid leukemia (AML) and meeting the diagnostic criteria for refractory AML are eligible, including: initial treatment cases with no response after two courses of standard therapy; relapse within 12 months after achieving complete remission (CR) and failure of consolidation therapy; relapse after 12 months with no response to conventional chemotherapy; two or more relapses; and presence of extramedullary leukemia.
- •Peripheral blood, bone marrow, or extramedullary lesion specimens showing leukemia cells with positive surface expression of EX02;
- •ECOG performance status score of 0-2;
- •Age ≥18 and ≤70 years, any gender;
- •Blood cell examination meeting the following conditions: hemoglobin > 60g/L, normal T lymphocyte count (CD3+) > 0.5×10^9/L, platelet count > 30×10^9/L;
- •Negative pregnancy test for women of childbearing potential before the start of the trial, and agreement to use effective contraception measures during the trial until the final follow-up; male participants of reproductive potential agreeing to use effective contraception measures during the trial until the final follow-up;
- •Voluntary participation in this clinical study: after fully understanding the study content, voluntary signing of the informed consent form, and commitment to complete all trial procedures and activities.
排除标准
- •Active hepatitis A, B, C, HIV infection, or other severe active infections that are not yet controlled;
- •History of acquired immune deficiency syndrome (AIDS) or long-term use of immunosuppressants due to other diseases (including steroids, equivalent to prednisone >15mg/day);
- •Conditions of the heart function: a. New York Heart Association (NYHA) class III or IV heart failure; b. Myocardial infarction or coronary artery bypass surgery within the six months prior to enrollment; c. Clinically significant ventricular arrhythmia or unexplained syncope; d. History of severe non-ischemic cardiomyopathy; e. History of cardiac dysfunction (left ventricular ejection fraction <45%) within 8 weeks before enrollment;
- •Pregnant or lactating women; participants (both male and female) unwilling to use contraception;
- •Severe liver or kidney dysfunction: aspartate aminotransferase (AST) and alanine aminotransferase (ALT) levels exceeding 3 times the upper limit of normal, bilirubin exceeding 3 times the upper limit of normal; serum creatinine ≥ 178 μmol/L (decompensated phase);
- •History of severe allergic reactions to any drugs planned for use in this study;
- •Recipients of hematopoietic stem cell transplantation must have discontinued immunosuppressants for at least 6 weeks before enrollment and have no signs of graft-versus-host disease;
- •Other conditions deemed unsuitable for participation in this trial by the investigator.
研究组 & 干预措施
CAR-T Cell Injection
干预措施: CAR-T Cell Injection (Biological)
结局指标
主要结局
Treatment-Emergent Adverse Events (TEAE)
时间窗: From the infusion (Day 0) to 3 months
TEAE is defined as any adverse event that occurs within 90 days after the first injection of EX02 CAR-T, or that existed before treatment and worsened after treatment. All adverse events are graded according to CTCAE v4.0, classified, and then the incidence rate is calculated. In addition, according to the definition of this study, the occurrence of CAR-T cell therapy-specific CRS and ICANS, as well as their grading, management, and prognosis are evaluated, and the incidence rates are calculated separately, with specific patient details recorded.
Overall Response Rate (ORR)
时间窗: From the infusion (Day 0) to Day 60
Overall response rate=number of complete remission(CR) (including incomplete hematologic recovery (CRi)) + number of partial remission (PR) / total number of treated cases, determined by the investigator according to the 2023 Chinese Diagnosis and Treatment Guidelines for Adult Acute Myeloid Leukemia (non-acute promyelocytic leukemia).
次要结局
- PFS(12 months)
- OS(12 months)
- ECOG scoring(2 months)
研究者
Zhimin Zhai
Chief of Hematology Department
The Second Hospital of Anhui Medical University