Anti-CD33 CAR-T Cells for the Treatment of Relapsed/Refractory CD33+ Acute Myeloid Leukemia
试验速览
- 阶段
- 1 期
- 发起方
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- The number and incidence of adverse events after anti-CD33 CAR infusion.
研究概览
简要总结
This is a phase I, interventional, single arm, open label, treatment study to evaluate the safety and tolerability of anti-CD33 CAR-T cells in patients with relapsed and/or refractory, high risk hematologic malignancies.
详细描述
AML is a rapidly progressing blood cancer and treated by high-dose multi-agent chemotherapy potentially followed by hematopoietic stem cell transplantation. Despite such intensive therapies, which are often associated with considerable toxicities and even death, about 60-70% of AML patients still relapse. Furthermore, the five-year survival rate from AML remains at a dismal 27%. AML is composed mostly of CD33+ leukemic blast cells. Therefore, CD33 is a potential good target by CAR T cells.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 60 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed written informed consent; Patients volunteer to participate in the clinical trial;
- •Diagnosis is mainly based on the World Health Organization (WHO) 2008;
- •Complete remission cannot be achieved after induction therapy; recurrence occurs after completion remission; the burden of leukemic blasts in the peripheral blood or bone marrow is greater than 5%;
- •Leukemic blast cells express CD33 (CD33 positive by flow cytometry or immunohistochemistry ≥70%);
- •The expected survival period is greater than 12 weeks;
- •ECOG score ≤2;
- •Age 2-60 years old;
- •HGB≥70g/L (can be transfused);
- •Total bilirubin does not exceed 3 times the upper limit of normal value, and AST and ALT do not exceed 5 times the upper limit of normal value.
排除标准
- •Patients declining to consent for treatment
- •Prior solid organ transplantation
- •One of the following cardiac issues: atrial fibrillation; myocardial infarction within the past 12 months; prolonged QT syndrome or secondary QT prolongation; clinically significant pericardial effusion; cardiac insufficiency NYHA (New York Heart Association) III or IV;
- •History of severe pulmonary dysfunction diseases;
- •Severe infection or persistent infection cannot be effectively controlled;
- •Severe autoimmune disease or congenital immunodeficiency;
- •Active hepatitis;
- •Human immunodeficiency virus (HIV) infection;
- •Clinically significant viral infections, or uncontrollable viral reactivation, including EBV (Epstein-Barr virus).
结局指标
主要结局
The number and incidence of adverse events after anti-CD33 CAR infusion.
时间窗: 1 year, particularly the first 3 months after CAR infusion
Determine the toxicity profile of anti-CD33 CAR T cell therapy including the number, incidence, and severity of symptoms such as cytokine release syndromes and neurotoxicity
次要结局
- Treatment-related mortality(one year after HCT)
- The disease response to anti-CD33 CAR T cells(4 weeks)
- Allogeneic hematopoietic stem cell transplantation (HCT)(42 days after HCT ingraftment)
- HCT 100% chymerism time(2 weeks after HCT)
- Overall survival(1 year after HCT)
- Progress-free survival(one year after HCT)