Statistical Analyses
death, melanoma, Toxicity, breast cancer
DISEASE PROGRESSION, MELANOMA, BREAST CANCER, EVENT, EVENTS
The planned sample size for this pilot study was up to 6 evaluable subjects with melanoma and up to 4 evaluable subjects with breast cancer. Subjects were evaluable for all endpoints if they received at least one dose of the RNA CAR T product infusion. If a subject consented but did not undergo cell infusion, that subject could be replaced. Toxicity was tabulated by grade and summarized using descriptive statistics, with patients with melanoma and breast cancer pooled for these analyses. After treatment of every 3 subjects, treatment-limiting events were calculated and an early pausing rule was established such that if significant evidence existed that the event rate of a TLT exceeded the maximum acceptable rate predefined in the protocol, the study should be suspended pending further evaluation. Overall response rate at day +25 (±5 days), progression-free survival (PFS) and overall survival (OS) were also analyzed and reported descriptively. PFS was defined as the time from the date of first infusion to the date of first documented disease progression or death due to any cause. OS was defined as the time from the date of first infusion to the date of death from any cause; if date of death was unavailable, OS was censored at the last date of contact.