A Phase II, Open Label, Multi-center Trial to Determine the Efficacy and Safety of Tisagenlecleucel Re-infusion in Pediatric and Adolescent Young Adult (AYA) Patients With Acute Lymphoblastic Leukemia Experiencing Loss of B Cell Aplasia
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 5
- 试验地点
- 4
- 主要终点
- Percentage of Patients Who Establish B Cell Aplasia Within 9 Months of Reinfusion
研究概览
简要总结
This was a multi-center Phase II study investigating the efficacy and safety of reinfusion of tisagenlecleucel in pediatric and young adult patients with acute lymphoblastic leukemia (ALL) who were treated with tisagenlecleucel and experience B cell recovery.
详细描述
This trial was a phase II, open label, multi-center trial to determine the efficacy and safety of tisagenlecleucel re-infusion in pediatric and adolescent young adult (AYA) patients with acute lymphoblastic leukemia (ALL) experiencing loss of B cell aplasia. Loss of B-cell aplasia is defined as: peripheral blood (PB) absolute B lymphocyte count ≥ 50/µL, OR PB B lymphocyte ≥ 10% of the total lymphocytes. B-cell aplasia is defined as PB absolute B lymphocyte count <50/µL.
The study had the following phases for all patients: Screening, Treatment and Follow-up. The total duration of the study was about 12 months. After tisagenlecleucel re-infusion, efficacy was assessed at months 1, 3, 6, and End of Study at which time blood samples were obtained.
The study stopped early due to slow enrollment into the trial. The rate of enrollment made the trial no longer feasible to continue.
The patients were able to voluntarily withdraw from the study for any reason, at any time. Patients who received commercial tisagenlecleucel had to be followed for up to 15 years post-infusion. Patients could have been followed under the Center for International Blood and Marrow Transplant Research (CIBMTR) cellular therapy registry if consented for participation. For patients who do not provide consent for participation in the Center for International Blood and Marrow Transplant Research (CIBMTR) registry, adverse events were to be reported for 15 years or until the patient enrolls in the registry.
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 25 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed informed consent must be obtained prior to participation in the study
- •Must have an additional dose of unexpired, commercial tisagenlecleucel available and prescribed by a physician in the course of medical practice
- •Age up to and including 25 years
- •Patients must have CD-19+ Leukemia
- •Patients who were previously treated with tisagenlecleucel and present with evidence of B-cell recovery as defined by: Peripheral blood (PB) absolute B lymphocyte count ≥ 50/µL, OR PB B lymphocyte ≥ 10% of the total lymphocytes
排除标准
- •Prior gene therapy other than tisagenlecleucel
- •Prior adoptive T cell therapy other than tisagenlecleucel
- •Active CNS involvement by malignancy
- •Active or latent hepatitis B or active hepatitis C, or any uncontrolled infection at screening
- •HIV positive test within 8 weeks of screening
研究组 & 干预措施
Tisagenlecleucel
Tisagenlecleucel Cell Dispersion for Infusion given once during the study.
The approved dose range for tisagenlecleucel is: 0.2 to 5.0×106 CAR positive viable T cells / kg for patients' ≤ 50 kg body weight or 0.1 to 2.5×108 CAR-positive viable T cells for patients > 50 kg body weight.
干预措施: Tisagenlecleucel (Biological)
结局指标
主要结局
Percentage of Patients Who Establish B Cell Aplasia Within 9 Months of Reinfusion
时间窗: Post-reinfusion up to 9 months (Day 1 is excluded)
Percentage of patients who establish B-cell aplasia at any visit following re-infusion with tisagenlecleucel. B-cell aplasia is defined as peripheral blood (PB) absolute B lymphocyte count \<50/μL. Planned timeframe was 12 months but actual timeframe was approximately 9 months due to early termination of the trial. Day 1 is post lymphodepleting chemotherapy and pre-reinfusion of tisagenlecleucel.
次要结局
- Overall Survival (OS)(Reinfusion up to 9 months)
- Complete Response (CR) or Complete Response With Incomplete Blood Count Recovery (CRi) by Day(Post-reinfusion up to 9 months)
- Participants With an Event(Reinfusion up to 9 months)