Statistical considerations
tumor
DISEASE, TUMOR
No formal power calculations were carried out. In the dose escalation phase, enrollment of approximately 54 patients was envisaged, with the actual number dependent on the number of dose escalation steps and the number of patients per cohort. In the dose expansion phase, enrollment of up to 16 patients in each of 8 cohorts was planned, representing an additional 128 patients. Patients were assigned to 1 of the 8 expansion cohorts based on tumor type, mutational status, and/or treatment history (Supplementary Table S1). An interactive voice response system (IVRS) was used to manage patient enrollment, cohort assignment and drug supplies.The safety population was defined as all patients who received any amount of tovorafenib. The DLT-evaluable population was defined as all patients in the dose escalation phase of the study who either experienced DLT during cycle 1 or who completed at least 75% of the planned doses and had sufficient follow-up data to allow the investigators and sponsor to determine whether DLT had occurred. The response-evaluable population included all patients with measurable disease who received any amount of tovorafenib and who had at least 1 postbaseline response assessment. The PK-evaluable population included all patients who had sufficient dosing data and concentration–time data to permit calculation of PK parameters.