Statistical considerations
death, tumour
TUMOUR
The sample size was calculated using a one-sample superiority test, function One Sample Proportion NIS of the Trial Size package of R software (version 3.6.3 [2020-02-29] “Holding the Windsock”. The R Foundation for Statistical Computing, Vienna, Austria). For Cohorts 1 to 3, according to previous reports, it was assumed that the 9-m CBR was 30% (null hypothesis) and a potential 20% increase was estimated with a superiority margin of 10% (alternative hypothesis). For cohort 4, according to previous reportsActivity analysis was based on the full analysis set (FAS) that included all enrolled patients. Safety was assessed for all patients who received at least one dose of study treatment. Data after the first progression for the three retreated patients was excluded from the current analysis. Continuous variables were summarised using descriptive statistics. Frequency counts and the percentage of subjects were provided for categorical data. Response rates were estimated using 95% confidence intervals (CI) or full range intervals. The survival or time-to-event endpoints were estimated using the Kaplan–Meier method. Cohen’s kappa test was used to compare the distribution of best objective responses and measures the agreement between RECIST and iRECIST assessment. Patients without documented progression or death at the time of the analysis were censored at the last date of tumour evaluation for PFS assessment. Patients without documented death at the time of the analysis were censored at the last date of follow-up for OS assessment. All statistical tests were considered two-tailed, and results with 