Long Term Follow-Up of Patients Exposed to Lentiviral-Based CAR T-Cell Therapy
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 1,400
- 试验地点
- 159
- 主要终点
- Percentage of patients with certain events (see description)
研究概览
简要总结
Per Health Authorities guidelines for gene therapy medicinal products that utilize integrating vectors (e.g. lentiviral vectors), long term safety and efficacy follow up of treated patients is required. The purpose of this study is to monitor all patients exposed to CAR-T therapied for 15 years following their last CAR-T (e.g. CTL019) infusion to assess the risk of delayed adverse events (AEs), monitor for replication competent lentivirus (RCL) and assess long-term efficacy, including vector persistence.
详细描述
Patients are enrolled following completion or early discontinuation from a Novartis sponsored or supported study of CAR T-Cell treatment. Patients will be followed for 15 years post treatment from the last treatment. They will be monitored for safety and efficacy within the primary treatment protocols for the protocol defined duration. Patients can drop off treatment protocols at any time to enter this long term Follow up study. Patients discontinuing from the primary treatment protocols for any reason will be enrolled in this long term follow up (LTFU). This will allow collecting data on long term safety and efficacy (as applicable) as mandated by the health authorities of all patients treated with CAR-T therapy within the concept of a single protocol.
Collection of such long term effects of CAR-T cell therapy will help to further define the risk-benefit profile of CAR-T Therapies.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 0 Years 至 100 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All patients who have received a CAR-T therapy and completed or discontinued early from a Novartis sponsored treatment protocol that utilized CAR-T cells or from any CAR-T trial sponsored by the University of Pennsylvania with which Novartis has a contractual agreement to co-develop the CAR technology.
- •Patients who have provided informed consent for the long term follow up study prior to their study participation .
排除标准
- •There are no specific exclusion criteria for this study.
结局指标
主要结局
Percentage of patients with certain events (see description)
时间窗: at M3 post treatment, M6, M9, M12 and then, every 6M up to year 5, yearly until year 15.
The percentage of pts with listed categories: New secondary malignancies, new serious infection, new incidence of serious neurologic disorder, New incidence or exacerbation of a prior rheumatologic or other autoimmune disorder, New incidence of a hematologic disorder
次要结局
- Percentage of patients who relapse or progress among patients who had not relapsed or progressed at study entry/re-entry;Incidence of death(at M3 post treatment then M6, M9, M12 and every 6M up to year 5, yearly until year 15.)
- B- and T- lymphocyte count(at M3 post treatment then M6, M9, M12 and every 6M up to year 5, yearly until year 15.)
- Percentage of patients with detectable CAR transgene levels in peripheral blood by q-PCR at pre- specified time points(at M3 post treatment, M6, M9, M12 and every 6M up to year 5, yearly until year 15.)
- Percentage of patients with detectable RCL by VSV-G(at M3 post treatment then M6, M9, M12 and every 6M up to year 5, yearly until year 15)
- Height and weight, Tanner staging, menstruation status(at M3 post treatment then M6, M12 and every year until year 15.)