Outcomes
tumor, DLTs
TUMOR
The primary objective was evaluation of safety and tolerability with endpoints including TEAEs, serious AEs, DLTs and detection of replication-competent lentivirus (RCL). Secondary endpoints included ORR confirmed by RECIST version 1.1, BOR, TTR, DoR, duration of SD, PFS and OS. Exploratory objectives included evaluation of cell persistence and cytokines. Exploratory endpoints included correlation of persistence, phenotype and functionality of transduced (afami-cel) and non-transduced T cells in the peripheral blood and/or tumor in response to treatment and safety; determination of target antigen expression, genes related to antigen processing/presentation and cell surface co-stimulatory ligands; and evaluation of serum cytokines (that is, IL-6). The following exploratory endpoints were not analyzed: evaluation of anti-tumor antibodies or candidate biomarkers from plasma-derived exosomes and cell-free DNA. Owing to the preliminary nature of the data collected before the data cutoff, limited correlative analyses were reported in relation to response to treatment and safety. Two patients received a second infusion; they were non-responders. Therefore, ORR was not evaluated for the second infusion.