Statistical methods
The study aimed to enroll approximately 35 patients, assuming a 10% dropout rate, to provide 32 patients for the analysis of IRC-assessed ORR. The full analysis set (FAS) was used for efficacy analyses, including all patients having received at least one dose of cabozantinib. The safety analysis set used for safety analyses was the same as the FAS. ORR was assessed as point estimates and two-sided 90% exact Clopper–Pearson CIs. The PFS and OS were estimated by Kaplan–Meier analysis. Safety was descriptively summarized. HRQoL values and changes from baseline were descriptively summarized. As an exploratory post hoc analysis, changes from baseline in plasma biomarker levels were evaluated over time at each time point. The differences in baseline plasma biomarker levels between responders and nonresponders were evaluated by two-sample 