Inefficiency of two-stage designs in phase II oncology clinical trials with high proportion of inevaluable patients. Contemp Clin Trials

Ji L, Whangbo J, Levine JE, Alonzo TA. Inefficiency of two-stage designs in phase II oncology clinical trials with high proportion of inevaluable patients. Contemp Clin Trials. 2022 Sep;120:106849. doi: 10.1016/j.cct.2022.106849. Epub 2022 Jul 19. PubMed PMID: 35868503; PubMed Central PMCID: PMC9489679.

Two-stage designs are commonly used for oncology Phase II clinical trials with a binary response endpoint. An issue that has not gained sufficient attention is the potential inefficiency in the usage of two-stage designs due to multiple enrollment suspensions when the proportion of patients inevaluable for response is high. Simulation studies were used to assess the performance of Simon’s two-stage designs, two-stage designs with a proposed modification, and a single-stage design in the context of Phase II clinical trials with a high proportion of patients inevaluable for response.