A Phase I/II Single Arm Study, Safety and Efficacy Assessment of the CD19 CAR T Cell on Pediatric Patients with Relapsing or Refractory B Cell Acute Lymphoblastic Leukemia (r/r B-ALL)
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 5
- 试验地点
- 1
- 主要终点
- Incidence of cytokine release syndrome: grade 3 and 4
研究概览
简要总结
The goal of this clinical trial is to evaluate the safety and efficacy of CD19 CAR-T cells in pediatric patients of all genders, aged 2 to 18 years, with relapsing or refractory B cell acute lymphoblastic leukemia (r/r B-ALL). The main questions it aims to answer are as following:
- What is the percentage of patients with overall remission rate (ORR) of complete response (CR) or complete remission with incomplete blood count recovery (CRi)?
- What is the rate of Event-free survival at first month and 2-3 months after intervention?
- What is the rate of Overall survival at first month and at 3 months after the intervention?
详细描述
B-cell acute lymphoblastic leukemia (B-ALL), as the most common type of pediatric tumor, is identified by unregulated cell proliferation of immature lymphoid cells that can infiltrate the bone marrow and blood. Also, relapse and refractory B-ALL (R/R B-ALL) is the main reason of global mortality due to the constraints of combination chemotherapy.
Over the past few years, substantial advancements have been made in treatment of ALL, specifically in the R/R context. Chimeric antigen receptor T (CAR-T) cells are a type of cancer immunotherapy treatment that function through modification of patient T cells to express CAR antigen on their surface. CAR-T cells aimed at CD19 have demonstrated promising activity in treatment of r/r B-ALL. In this study we aim to evaluate safety and efficacy of Anti-CD19 CAR T cell therapy in children with R/R B-ALL.
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
- 2 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Ages 2 to 18 years with relapsed or refractory CD19+ B-ALL
- •Presence of disease in the bone marrow
- •Able to tolerate the apheresis process
- •Life expectancy > 12 weeks
- •Lansky or Karnofsky score > 50%
- •At least 7 days passed since the last chemotherapy and the last treatment with corticosteroids
- •Informed consent
- •Having potential donor for stem cell transplantation
排除标准
- •Presence of active malignancy other than the disease under study
- •Chloroma and leukemic infiltration on MRI or significant neurological symptoms
- •Any CNS disorder
- •Presence of active GVHD
- •Radiation therapy within last 14 days
- •History of Anti-CD19 or Anti-CD20 therapy
- •Donor lymphocyte injection or other cell therapy methods within the last 30 days
- •Presence of severe active infection
- •Organ dysfunction
研究组 & 干预措施
Anti-CD19 CAR-T treatment group
干预措施: anti-CD19 CAR T cell therapy (Biological)
结局指标
主要结局
Incidence of cytokine release syndrome: grade 3 and 4
时间窗: First month and 3 months after intervention
Percentage of patients with overall remission rate (ORR) of complete response (CR) or complete remission with incomplete blood count recovery (CRi)
时间窗: First month and 2-3 months after intervention
Overall survival
时间窗: First month and 3 months after intervention
Incidence of Immune effector cell-associated neurotoxicity syndrome (ICANS): grade 3 and 4
时间窗: First month and 3 months after intervention
Event-free survival
时间窗: First month and 2-3 months after intervention
次要结局
- Percentage of patients with overall remission rate (ORR) of complete response (CR) or complete remission with incomplete blood count recovery (CRi)(6 months and 12 months after intervention)
- Investigation of Minimal residual disease in patient(First month and 2-3 months after intervention)
- Incidence of cytokine release syndrome: grade 3 and 4(6 months and 12 months after intervention)
- Incidence of Immune effector cell-associated neurotoxicity syndrome (ICANS): grade 3 and 4(6 months and 12 months after intervention)
- Incidence of tumor lysis syndrome (TLS)(Months 1, 3, 6, and 12 after the intervention)
- Incidence of leukopenia(Months 1, 3, 6, and 12 after the intervention)
- Incidence of infection(Months 1, 3, 6, and 12 after the intervention)
- Event-free survival(6 months and 12 months after intervention)
- Overall survival(6 months and 12 months after intervention)