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Activity Number: 46 - Recent Developments in Novel Clinical Trial Design and Analysis for Precision Medicine
Type: Invited
Date/Time: Sunday, July 28, 2019 : 4:00 PM to 5:50 PM
Sponsor: ENAR
Abstract #300008 Presentation
Title: Bayesian Clinical Trial Designs to Evaluate Subgroup-Specific Treatment Effects
Author(s): Peter Thall* and Juhee Lee and Thomas Murray and Andrew Chapple
Companies: U.T. M.D. Anderson Cancer Center and University of California, Santa Cruz and University of Minnesota and Louisiana State University
Keywords: Clinical trial design; Utility; Precision medicine; Bayesian design; Cancer clinical trial

This talk will present three Bayesian clinical trial designs in which outcome adaptive decisions are made within predefined prognostic subgroups. The first is a randomized trial comparing nutritional prehabilitation to standard of care for controlling post-operative morbidity (POM) following chemoradiation and esophageal resection. The design accounts for two prognostic subgroups while assuming a robust generalization of proportional odds model for POM as a function of treatment and subgroup. The second is a phase I design to optimize dose based on time to toxicity that generalizes the time-to-event continual reassessment method, while allowing adaptive combination of subgroups having similar dose-toxicity profiles. The third is a phase I-II trial of natural killer cells for advanced hematologic malignancies that does dose optimization and sequential safety monitoring within six prognostic subgroups, based on five time-to-event outcomes. All designs are shown by simulation to be greatly superior to their conventional comparators.

Authors who are presenting talks have a * after their name.

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