Clinical Study of the Safety and Efficacy of Non-viral Site-directed Integrated PD1-BCMA-CART in Adult Treatment of Relapsed or Refractory Multiple Myeloma
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 9
- 试验地点
- 1
- 主要终点
- Objective response rate (ORR)
研究概览
简要总结
This trial aims to evaluate the safety and efficacy of PD1-BCMA-CART in treating patients with relapsed or refractory multiple myeloma.
详细描述
Using gene editing, chimeric antigen receptors recognizing BCMA were integrated into subject self-derived T cells to obtain a large number of BCMA-CART by in vitro amplification, and BCMA-CART back into the subjects could identify and kill myeloma cells in the subjects.This open-label, dose-escalation study was designed to evaluate the safety and antitumor efficacy of PD1-BCMA-CART in the treatment of relapsed or refractory multiple myeloma.
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
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Have the capacity to give informed consent;
- •Confirmed diagnosis of active MM as defined by NCCN and IMWG criteria;
- •Have a diagnosis of BCMA+ multiple myeloma (MM), (≥ 5% BCMA+ in CD138+ plasma cells by flow cytometry obtained within 45 days of study enrollment);
- •Refractory and relapsed MM patients after > 2 cycles of induction therapy,or,have relapsed or treatment refractory disease following autologous stem cell transplant (ASCT);
- •ECOG score=0-
- •Subjects according with any of the following options:
- •Failure with separation of T cells during autologous CART processing; or,
- •Failure with expansion of autologous CART; or,
- •The proportion of T cells in PBMC <10%; or,
- •Won't benefit from autologous CART therapy because of disease progress.
排除标准
- •Pregnant or nursing women; Women of reproductive potential must have a negative serum pregnancy test performed within 48 hours of starting conditioning chemotherapy
- •Active infection, HIV infection, syphilis serology reaction positive;
- •Active hepatitis B, hepatitis C at the time of screening
- •Significant hepatic dysfunction as following, SGOT(serum glutamic-oxaloacetic transaminase)> 5 x upper limit of normal; bilirubin > 3.0 mg/dL;
- •Lymphotoxic chemotherapeutic agents within 2 weeks of leukapheresis
- •serious mental disorder;
- •With severe cardiac, liver, renal insufficiency, diabetes and other diseases;
- •Participate in other clinical research in the past three months; previously treatment with any gene therapy products
- •Contraindication to cyclophosphamide or fludarabine chemotherapy
结局指标
主要结局
Objective response rate (ORR)
时间窗: Up to 90 days after T cell infusion
Proportion of patients in whom a response among complete response or partial response, as defined by International Myeloma Working group(IMWG) response criteria , will be observed.
次要结局
- Duration of persistence of PD1-BCMA-CART(Baseline up to 2 year)
- Incidence and Severity of Adverse Events as a Measure of Safety and Tolerability(Up to 35 days after T cell infusion)