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).
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.
Study Type
OBSERVATIONAL
Enrollment
45
CT (Computerized Tomography) scan or PET-CT (Positron Emission Tomography-Computerized Tomography) scans as per protocol
Tumor Biopsy per protocol
Blood Test per protocol
Massachusetts General Hospital
Boston, Massachusetts, United States
RECRUITINGOverall Survival
defined as the time from randomization (or registration) to death due to any cause, or censored at date last known alive
Time frame: 1 month
Overall Survival
defined as the time from randomization (or registration) to death due to any cause, or censored at date last known alive
Time frame: 6 months
Overall Survival
defined as the time from randomization (or registration) to death due to any cause, or censored at date last known alive
Time frame: 12 months
Overall Survival
defined as the time from randomization (or registration) to death due to any cause, or censored at date last known alive
Time frame: 24 months
Progresison Free Survival
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.
Time frame: 1 month
Progresison Free Survival
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.
Time frame: 6 months
Progresison Free Survival
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.
Time frame: 12 months
Progresison Free Survival
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.
Time frame: 24 months
New incidence or exacerbation of a pre-existing neurologic disorder or prior rheumatologic or other autoimmune disorder
An annual physical exam will be conducted for surveillance of manifestations indicative of oncoretroviral diseases.
Time frame: Up to 15 Years
New incidence of a hematologic disorder
An annual physical exam will be conducted for surveillance of manifestations indicative of oncoretroviral diseases.
Time frame: up to 15 years
Number of Participants with Treatment-Related Adverse Events as Assessed by CTCAE v 5.0
NCI Common Terminology Criteria for Adverse Events (CTCAE)
Time frame: up to 15 years
Vector-derived RCL
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).
Time frame: baseline and in 3 months, 6 months, and annually up to 15 years
Assessment of CAR T cells persistence by VCN in peripheral blood
VCN will be performed in DNA from whole blood to monitor for persistence of vector sequence
Time frame: baseline and in 3 months, 6 months, every 6 months up to 5 years, and annually up to 15 years
Progression-free Survival
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.
Time frame: 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
Overall Survival
The time from post-CAR T cells infusion to the date of death.
Time frame: 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
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