Statistical Analysis
PD, death
DISEASE
The planned enrollment was estimated to be 6–9 evaluable patients in phase I and 20–38 evaluable patients in phase II. For the primary endpoint of ORR, a Simon two-stage design was used to test the null hypothesis that ORR ≤ 0.20 versus the alternative that ORR ≥ 0.50. After enrollment of 20 evaluable patients in the first stage, the trial would be terminated if 2 or fewer patients respond. If the trial continues to the second stage, a total of 38 patients would be studied. Patients from the phase I dose escalation who were treated at the RP2D were included in the efficacy assessments in Simon stage 1 of the phase II dose expansion.The ORR, along with a 95% confidence interval (CI), was estimated. The PFS and OS survival curves and medians were estimated using the Kaplan–Meier method and reported with the corresponding two-sided 95% Brookmeyer–Crowley CI, from the on-treatment date to progressive disease (PD), death, or data cut-off date. All statistical analyses were performed by an independent statistician to ensure unbiased data review and conducted on R version 4.1.0, R Core Team (2021).