biostudies-literatureQiu YNCI NIH HHSNIH HHS214https://www.ebi.ac.uk/biostudies/studies/S-EPMC12466004biostudies-literatureUnknown25(1)<h4>Background</h4>Combination therapies involving novel agents, such as immunotherapies and targeted therapies, offer significant antitumor benefits by increasing dose intensity, targeting multiple pathways, and benefiting a broader patient population. To further explore these advantages, the National Cancer Institute (NCI) has initiated Combination Therapy Platform Trial with Molecular Analysis for Therapy Choice (ComboMATCH) to evaluate the effectiveness of new drug combinations in treating both adults and children. However, designing dose optimization trials for these combination therapies presents substantial challenges due to the complex interactions and unique mechanisms of action.<h4>Methods</h4>To address these challenges, we propose COMPACT, a Bayesian phase I-II randomized desigBMC medical research methodologyA randomized Bayesian phase I-II dose optimization design for combination cancer therapies with progression-free survival end point.PMC12466004P30CA142543P30 CA142543Qiu YLi MfalseA randomized Bayesian phase I-II dose optimization design for combination cancer therapies with progression-free survival end point.<h4>Background</h4>Combination therapies involving novel agents, such as immunotherapies and targeted therapies, offer significant antitumor benefits by increasing dose intensity, targeting multiple pathways, and benefiting a broader patient population. To further explore these advantages, the National Cancer Institute (NCI) has initiated Combination Therapy Platform Trial with Molecular Analysis for Therapy Choice (ComboMATCH) to evaluate the effectiveness of new drug combinations in treating both adults and children. However, designing dose optimization trials for these combination therapies presents substantial challenges due to the complex interactions and unique mechanisms of action.<h4>Methods</h4>To address these challenges, we propose COMPACT, a Bayesian phase I-II randomized desig2025-01-01T00:00:00Z2025 Sep2026-06-03T21:45:53.818Z2026-05-02T03:08:44.94ZS-EPMC124660044101326510.1186/s12874-025-02665-4