Long-term Follow-up of Subjects Treated With CAR T Cells
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 45
- 试验地点
- 2
- 主要终点
- Overall Survival
研究概览
简要总结
This is a single site, non-randomized, open-label, long-term safety and efficacy follow-up study for Phase 1 studies that evaluate the safety and efficacy of CAR T cells: NCT05660369 (DF/HCC# 22-175) and NCT06026319 (DF/HCC# 23-474).
详细描述
This is a long-term safety and efficacy follow-up study for subjects who have been treated with CAR T cells in corresponding Phase I main studies that evaluated the safety and efficacy of CAR T cells in subjects.
No investigational treatment will be administered in this study.
The FDA (2020) recommends long-term follow up for subjects treated with gene therapy drug products to monitor for delayed adverse events (AEs), as well as durability of clinical response.
Therefore, after monitoring of subjects in the main studies has been completed (24 months after CAR T cells infusion, or <24 months after CAR T cells infusion if subject terminated early due to disease progression or due to discontinuing corresponding main study for any reason), subjects will be asked to participate in a long-term follow-up study (LTFU).
Subjects will be followed for up to 15 years following their last CAR T cells infusion in the corresponding main study.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects will be asked to participate leading up to the last DF/HCC corresponding main study visit.
- •Subjects meeting the following criteria are eligible for study participation:
- •Provision of voluntary written informed consent by subject
- •CAR T cells were administered in DF/HCC IRB corresponding main study
排除标准
- •Subjects meeting the following criterion are to be excluded from study participation:
- •- Subject unable to comply with study requirements
研究组 & 干预措施
CAR T cells
- Eligibility to participate on this study if enrolled on a DF/HCC IRB corresponding main study and received infusion of CAR T cells
- The research study procedures include evaluations and follow up visits: Timepoints of each evaluation and follow up visit- per protocol
- Medical History/Physical Exam
- Blood Test
- Assessment of Disease: CT (Computerized Tomography) scan or PET-CT (Positron Emission Tomography-Computerized Tomography) scans.
- Tumor biopsy.
- Data Collection
- Biobanking
干预措施: Disease assessments (Diagnostic Test)
CAR T cells
- Eligibility to participate on this study if enrolled on a DF/HCC IRB corresponding main study and received infusion of CAR T cells
- The research study procedures include evaluations and follow up visits: Timepoints of each evaluation and follow up visit- per protocol
- Medical History/Physical Exam
- Blood Test
- Assessment of Disease: CT (Computerized Tomography) scan or PET-CT (Positron Emission Tomography-Computerized Tomography) scans.
- Tumor biopsy.
- Data Collection
- Biobanking
干预措施: Tumor Biopsy (Procedure)
CAR T cells
- Eligibility to participate on this study if enrolled on a DF/HCC IRB corresponding main study and received infusion of CAR T cells
- The research study procedures include evaluations and follow up visits: Timepoints of each evaluation and follow up visit- per protocol
- Medical History/Physical Exam
- Blood Test
- Assessment of Disease: CT (Computerized Tomography) scan or PET-CT (Positron Emission Tomography-Computerized Tomography) scans.
- Tumor biopsy.
- Data Collection
- Biobanking
干预措施: Blood test (Diagnostic Test)
结局指标
主要结局
Overall Survival
时间窗: From post-CAR T cells infusion date until documented date of death from any cause, assessed post treatment every 6 months through 5 years and then annually up to 15 years
The time from post-CAR T cells infusion to the date of death.
Progresison Free Survival
时间窗: 24 months
PFS is defined as the number of days from the day of CAR T cells treatment to the first documented disease progression or date of death, whichever occurs first. Patients who are lost to follow up without a known date of progression or death due to any cause will be censored in the analysis at the date of their last available tumor assessment.
New incidence or exacerbation of a pre-existing neurologic disorder or prior rheumatologic or other autoimmune disorder
时间窗: Up to 15 Years
An annual physical exam will be conducted for surveillance of manifestations indicative of oncoretroviral diseases.
New incidence of a hematologic disorder
时间窗: up to 15 years
An annual physical exam will be conducted for surveillance of manifestations indicative of oncoretroviral diseases.
Number of Participants with Treatment-Related Adverse Events as Assessed by CTCAE v 5.0
时间窗: up to 15 years
NCI Common Terminology Criteria for Adverse Events (CTCAE)
Vector-derived RCL
时间窗: baseline and in 3 months, 6 months, and annually up to 15 years
Archiving of samples for potential detection of vector-derived RCL. RCL testing will be monitored at the central RCL lab (Indiana University) by a suitable qPCR assay for detection of the lentiviral vector (VSV-g DNA).
Assessment of CAR T cells persistence by VCN in peripheral blood
时间窗: baseline and in 3 months, 6 months, every 6 months up to 5 years, and annually up to 15 years
VCN will be performed in DNA from whole blood to monitor for persistence of vector sequence
Progression-free Survival
时间窗: From post-CAR T cells infusion date until date of first documented disease progression or date of death from any cause, assessed up to 15 years post treatment
The number of days from the day of CAR T cells treatment to the first documented disease progression or date of death, whichever occurs first. Patients who are lost to follow up without a known date of progression or death due to any cause will be censored in the analysis at the date of their last available tumor assessment.
Overall Survival
时间窗: 6 months
defined as the time from randomization (or registration) to death due to any cause, or censored at date last known alive
Overall Survival
时间窗: 1 month
defined as the time from randomization (or registration) to death due to any cause, or censored at date last known alive
Overall Survival
时间窗: 12 months
defined as the time from randomization (or registration) to death due to any cause, or censored at date last known alive
Overall Survival
时间窗: 24 months
defined as the time from randomization (or registration) to death due to any cause, or censored at date last known alive
Progresison Free Survival
时间窗: 1 month
PFS is defined as the number of days from the day of CAR T cells treatment to the first documented disease progression or date of death, whichever occurs first. Patients who are lost to follow up without a known date of progression or death due to any cause will be censored in the analysis at the date of their last available tumor assessment.
Progresison Free Survival
时间窗: 6 months
PFS is defined as the number of days from the day of CAR T cells treatment to the first documented disease progression or date of death, whichever occurs first. Patients who are lost to follow up without a known date of progression or death due to any cause will be censored in the analysis at the date of their last available tumor assessment.
Progresison Free Survival
时间窗: 12 months
PFS is defined as the number of days from the day of CAR T cells treatment to the first documented disease progression or date of death, whichever occurs first. Patients who are lost to follow up without a known date of progression or death due to any cause will be censored in the analysis at the date of their last available tumor assessment.
次要结局
未报告次要终点
研究者
Marcela V. Maus, M.D.,Ph.D.
Sponsor Investigator
Massachusetts General Hospital