Phase 1/2 Study of Anti-CD33 Chimeric Antigen Receptor-Expressing T Cells (CD33CART) in Children and Young Adults With Relapsed/Refractory Acute Myeloid Leukemia
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 52
- 试验地点
- 6
- 主要终点
- Morphologic remission
研究概览
简要总结
This phase 1/2 trial aims to determine the safety and feasibility of antiCD33 chimeric antigen receptor (CAR) expressing T cells (CD33CART) in children and adolescents/young adults (AYAs) with relapsed/refractory acute myeloid leukemia (AML). The trial will be done in two phases: Phase 1 will determine the maximum tolerated dose of CD33CART cells using a 3+3 trial design, with dose-escalation for autologous products separated from dose-escalation for an allogeneic arm. Phase 2 is an expansion phase designed to evaluate the rate of response to CD33CART.
详细描述
This study consists of two phases. The objectives of Phase 1 and Phase 2 are:
Phase 1:
Autologous Arm: To determine the maximum tolerated dose of lentivirally transduced autologous CD33-redirected CAR-T cells (CD33CART) in children and young adults with relapsed/refractory AML
Allogeneic Arm: To determine the maximum tolerated dose of lentivirally transduced allogeneic CD33-redirected CAR-T cells (ALLO-CD33CART) in children and young adults with post-HSCT relapsed/refractory AML
Phase 2:
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 35 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
CD33CART autologous
Patients who receive an autologous CD33CART cell infusion
干预措施: CD33CART autologous (Biological)
CD33 CART allogeneic
Patients who receive an allogeneic CD33CART cell infusion
干预措施: CD33CART allogeneic (Biological)
结局指标
主要结局
Morphologic remission
时间窗: Day 28 post CD33CART infusion
To determine the percentage of recipients treated with CD33CART who achieve morphologic remission (\<5% blasts in marrow) at Day 28 post-CD33CART cell infusion
Maximum tolerated dose - Autologous Arm
时间窗: Day 28 post CD33CART infusion
To determine the maximum tolerated dose of lentivirally-transduced autologous CD33-redirected CAR-T cells (CD33CART) in children and young adults with relapsed/refractory AML
Maximum tolerated dose - Allogeneic Arm
时间窗: Day 28 post CD33CART infusion
To determine the maximum tolerated dose of lentivirally-transduced allogeneic CD33-redirected CAR-T cells (ALLO-CD33CART) in children and young adults with post-HSCT relapsed/refractory AML
次要结局
- Feasibility of CD33CART infusion(6 weeks post apheresis)
- Molecular Cytokine release syndrome (CRS), sinusoidal occlusion syndrome (SOS), or other CD33CART related toxicities(8 weeks post CD33CART infusion)
- Overall survival, event-free survival and treatment-related mortality(28 days post CD33CART infusion)
- GVHD(30 days post CD33CART infusion)
- Allogeneic hematopoietic stem cell transplantation(6 weeks post CD33CART infusion)
- Feasibility of CD33CART manufacture(2 weeks post start of CD33CART manufacture)
- Morphologic remission(28 days post CD33CART infusion)
- MRD negativity(28 days post CD33CART infusion)
- SOS and other post-transplant toxicities(6 weeks post HCT)
- Post-HCT time to engraftment(6 weeks post HCT)
- Molecular remission(28 days post CD33CART infusion)