Adaptive Two-Stage Designs for One-Arm Binary Endpoint in Oncology Clinical Trials

• Activity Number: 153 - Contributed Poster Presentations: Biopharmaceutical Section
• Type: Contributed
• Date/Time: Monday, August 8, 2022 : 10:30 AM to 12:20 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Yu Zhao* and Weichao Bao and Kui Shen
• Companies: University of California, San Diego and Bayer U.S. LLC and Bayer U.S. LLC
• Keywords: Adaptive Design; Clinical Trial; Binary Endpoint

Abstract

Biomarker guided clinical trial designs have played a crucial role in the precision medicine strategy during drug development. Although various designs have already been proposed, it remains very challenging to identify the population to be evaluated in pivotal phase II/III trials for targeted therapies, specifically, in identifying a subgroup at the interim or final analysis for further evaluation. This research investigated three separate adaptive two-stage designs (1.Jone, 2.Parashar, and 3.Tournoux) for a binary endpoint, such as objective response rate (ORR), by incorporating biomarker status into the design. The methods of the three designs were further improved for parameter search with both type I and type II error controls. Lastly, the improved designs were compared with the traditional Simon’s two-stage design in terms of the sample size, type I error, and type II error.