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Activity Number: 31
Type: Contributed
Date/Time: Sunday, August 4, 2013 : 2:00 PM to 3:50 PM
Sponsor: Biopharmaceutical Section
Abstract - #309582
Title: An 'Early-Go' Design for Single-Arm, Two-Stage, Phase II Clinical Trials
Author(s): William E. Brady*+ and Gregory Wilding
Companies: Roswell Park Cancer Institute and Roswell Park Cancer Institute
Keywords: phase II clinical trial ; two-stage design ; exact calculations
Abstract:

We propose a new, single-arm, two-stage phase II clinical trial using a tumor response as the primary endpoint. We introduce an 'early-go' decision--based on increasing disease (i.e., early progression)--to a two-stage design, in which the decision to stop for futility at the first stage and the decision regarding the compound's activity at the second stage are based on complete or partial tumor response. In typical two-stage designs, lengthy suspensions are common while the first stage is reviewed. We incorporate increasing disease into an 'early-go' decision--prior to the end of the first stage--to allow the trial to go to the second stage if sufficient responses or a sufficiently small number of patients with increasing disease are observed. This can potentially shorten the duration of the trial by reducing or eliminating the delay between the first and second stages of accrual. However, the utility of increasing disease for inference is not clear, so inference is based solely on response. Exact calculations are shown for the operating characteristics of the trial and are compared to the Simon (1989) design, and the expected trial durations for the designs are compared.


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