Primary analysis and expansion cohorts
tumor
TUMOR
The primary analysis cohort enrolled a maximum of 25 patients per tumor type. Multiple interim analyses (every 12 weeks) were performed to monitor the safety and efficacy and to determine whether a cohort should discontinue enrollment early because of success or futility. Response data for a minimum of five patients in a given cohort were required before discontinuation due to futility (<30% probability of exceeding the historical response rate), and a minimum of 10 patients were required before discontinuation for efficacy (>95% probability of exceeding the historical response rate). If a cohort closed early for efficacy, a histology subtype-specific expansion cohort, designed to provide supportive efficacy data, could be opened to accommodate additional patient enrollment. Expansion cohorts did not contribute to the Bayesian modeling, and there was no specific sample size calculation for these cohorts. Investigators were allowed to enroll patients into the expansion cohort for the duration of trial enrollment. At the final analysis, enrollment of a minimum of two patients in a given cohort was required to meet statistical significance.