Sample size considerations
toxicities, DLTs
PAD
Part 1 dose escalation used a 3 + 3 + 3 design, and the sample size was determined by the frequency of dose-limiting toxicities (DLTs) and final number of dose levels tested before the MTD or PAD was established.For Part 2 expansion, the sample size was based on the Simon 2-stage design. Based on a one-sided type I error of 0.05 and 80% power, a total of 27 patients with 8 subjects in Stage 1 would be required to demonstrate the desired response rate of 20% assuming the response rate for the historical control was 3%.