A Phase I Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of CLL1-/CD33 Targeted LCAR-AMDR Cells Product in Patients With Relapsed/Refractory Acute Myeloid Leukemia
概览
- 阶段
- 1 期
- 干预措施
- LCAR-AMDR Cells Product
- 疾病 / 适应症
- Acute Myeloid Leukemia
- 发起方
- Institute of Hematology & Blood Diseases Hospital, China
- 入组人数
- 4
- 试验地点
- 2
- 主要终点
- CAR positive T cells and CAR transgene levels in peripheral blood and bone marrow
- 状态
- 终止
- 最后更新
- 2个月前
概览
简要总结
This is a prospective, single-arm, open-label, single dose-finding and dose-expansion study that evaluates the safety, tolerability, PK, and anti-tumor efficacy of LCAR-AMDR cells in subjects with relapsed/refractory Acute Myeloid Leukemia who received adequate standard therapy.
研究者
入排标准
入选标准
- •The subject voluntarily participates in the clinical study; Fully understand and be Informed of the study and sign the Informed consent (Informed Consent Form, ICF)（For minors, the guardian shall also provide written informed consent ）; Willing to follow and able to complete all test procedures; Informed consent must be obtained before initiating any tests or procedures related to the study that are not part of the standard treatment of the subject's disease;
- •Age 14-60 years;
- •ECOG score: ≤2;
- •Relapsed/refractory AML must meet one of the following conditions:
- •Twice or more relapse;
- •Newly diagnosed AML patients who failed after 2 cycles of standard chemotherapy;
- •Relapse within 12 months after CR, or relapse after 12 months with CR but failed to respond to conventional chemotherapy;
- •Persistent extramedullary leukemia.
- •Meet the requirements of allogeneic HSCT
- •Expected survival ≥ 3 months;
排除标准
- •Subject with APL/AML-M3:t(15;17)(q22;q12)
- •Received any of the following treatments:
- •Previous allo-HSCT(Subjects who received allo-HSCT for more than 6 months, have stopped immunosuppressive drugs and have no active GvHD are not included in the exclusion criteria)
- •Previous gene therapy
- •Previous anti CD33/CLL1 therapy
- •Previous any target CAR-T cells therapy
- •Prior antitumor therapy with insufficient washout period;
- •CNS infiltration; Except for patients with prior CNS infiltration who are currently in remission;
- •HBsAg, HBV DNA, HCV-Ab, HCV RNA or HIV-Ab positive;
- •Pregnant or breast-feeding women;
研究组 & 干预措施
LCAR-AMDR Cells Product
Each subject will be treated with LCAR-AMDR Cells
干预措施: LCAR-AMDR Cells Product
结局指标
主要结局
CAR positive T cells and CAR transgene levels in peripheral blood and bone marrow
时间窗: 2 years after LCAR-AMDR infusion (Day 1)
CAR positive T cells and CAR transgene levels in peripheral blood and bone marrow after LCAR-AMDR infusion
Recommended Phase 2 dose (RP2D) finding
时间窗: 30 days after LCAR-AMDR infusion (Day 1)
RP2D established through ATD+BOIN design
Incidence, severity, and type of treatment-emergent adverse events (TEAEs)
时间窗: Time Frame: Minimum 2 years after LCAR-AMDR infusion (Day 1)
An adverse event refers to any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product (investigational or non-investigational), which does not necessarily have a causal relationship with the treatment
次要结局
- Event-free survival (EFS)(Minimum 2 years after LCAR-AMDR infusion (Day 1))
- The proportion of subjects who achieve CR or Cri and obtain bone marrow MRD negative.(Minimum 2 years after LCAR-AMDR infusion (Day 1))
- Incidence of anti-LCAR-AMDR antibody and positive sample titer(Minimum 2 years after LCAR-AMDR infusion (Day 1))
- Overall Survival (OS)(Minimum 2 years after LCAR-AMDR infusion (Day 1))
- Time to Response (TTR)(2 years after LCAR-AMDR infusion (Day 1))
- Duration of Response (DoR)(Minimum 2 years after LCAR-AMDR infusion (Day 1))
- Overall response rate (ORR)(2 years after LCAR-AMDR infusion (Day 1))