Prospective Evaluation Of Delayed Effects Of Pediatric Car T Cell Therapy (PROSPER)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 100
- 试验地点
- 8
- 主要终点
- Presence of bone marrow dysfunction (BMD)
研究概览
简要总结
This study is being done to learn more about the short-term and long-term side effects of CAR-T cell therapy. Specifically, researchers want to know how often patients get infections, have delays in recovering blood cell counts and/or have damage to the nervous system.
详细描述
Primary Objectives
- Bone Marrow Function: To report on the incidence, timing, severity of, and risk factors for bone marrow dysfunction in participants in remission or without bone marrow involvement of disease at 3- and 6-months following CAR T cell therapy. (B-ALL cohort)
- Infection/Immune Reconstitution: To evaluate the incidence, timing, severity of and risk factors for clinically significant infections following CAR T cell therapy at 3- and 6-months following CAR T cell therapy. (B-ALL cohort)
- Neurotoxicity: To evaluate the incidence, timing, severity of, and risk factors for persistent ICANS at 3- and 6-months post CAR T cell therapy. (B-ALL cohort)
Secondary Objectives
- To evaluate bone marrow function, infection/immune reconstitution, and neurotoxicity at 12 months and 24 months post CAR T cell therapy in participants with B-ALL.
- To characterize bone marrow function, infection/immune reconstitution, and neurotoxicity between 3 and 24 months after CAR T cell therapy in other hematologic malignancies and solid tumor cohorts.
Participants will have an assessment of preexisting morbidity and potential risk factors, collection of specimens for banking, scheduled late effects monitoring, laboratory analysis, and screening studies. Data and biospecimens will be collected at 3 months, 6 months, 1 year and 2 years after CAR T cell infusion.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- — 至 30 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants must have received an initial systemically-administered CAR T cell infusion within the last 1-3 months (+/- 14 days).
- •Initial infusion is defined as the first administration of a CAR T cell product the participant has not previously received OR receipt of a CAR T cell product previously received after an interval allogeneic HSCT.
- •Age ≤ 30 years at CAR T cell infusion.
排除标准
- •Active malignancy other than the disease under study.
- •Planned consolidative HSCT within 3 months post CAR T cell infusion.
- •Received or planned additional disease directed therapy post CAR T cell infusion.
- •Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.
研究组 & 干预措施
Other hematologic malignancy
Other hematologic malignancy participants who have received initial CAR T cell therapy within the last 1-3 months.
Solid tumor (ST)
ST participants who have received initial CAR T cell therapy within the last 1-3 months.
B cell acute lymphoblastic leukemia (B cell acute lymphoblastic leukemia (B-ALL) ) cohort
B-ALL participants who have received initial CAR T cell therapy within the last 1-3 months.
结局指标
主要结局
Presence of bone marrow dysfunction (BMD)
时间窗: Within 6 months post CAR T-cell therapy
Among patients in remission or without bone marrow involvement of disease in the B-ALL cohort, we will summarize the rates of prevalent BMD at 3- and 6-months post-infusion and estimate the cumulative incidence of new BMD and BMD recovery for patients with prevalent BMD at 3- and 6-months.
Occurrence of clinically significant infections
时间窗: Within 6 months post CAR T-cell therapy
The infection density of clinically significant infections in 3-6 months will be summarized in the B-ALL cohort.
Presence of persistent ICANS
时间窗: Within 6 months post CAR T-cell therapy
We will summarize the rates of persistent ICANS at 3- and 6-months post-infusion and estimate the cumulative incidence and timing of new ICANS and ICANS recovery for patients with persistent ICANS at 3- and 6-months in the B-ALL cohort.
次要结局
- Severity of clinically significant infections(Within 24 months post CAR T-cell therapy)
- Presence of bone marrow dysfunction (BMD)(Within 24 months post CAR T-cell therapy)
- Severity of BMD(Within 24 months post CAR T-cell therapy)
- Occurrence of clinically significant infections(Within 24 months post CAR T-cell therapy)
- Time to the earliest clinically significant infection(Within 24 months post CAR T-cell therapy)
- Presence of persistent ICANS(Within 24 months post CAR T-cell therapy)
- Severity of persistent ICANS(Within 24 months post CAR T-cell therapy)