Two-stage design for phase II oncology trials with relaxed futility stopping.

TitleTwo-stage design for phase II oncology trials with relaxed futility stopping.
Publication TypeJournal Article
Year of Publication2016
AuthorsIvanova, Anastasia, and Allison M. Deal
JournalStat Interface
Volume9
Issue1
Pagination93-98
Date Published2016
ISSN1938-7989
Abstract

Many oncology phase II trials are single arm studies designed to screen novel treatments based on efficacy outcome. Efficacy is often assessed as an ordinal variable based on a level of response of solid tumors with four categories: complete response, partial response, stable disease and progression. We describe a two-stage design for a single-arm phase II trial where the primary objective is to test the rate of tumor response defined as complete plus partial response, and the secondary objective is to estimate the rate of disease control defined as tumor response plus stable disease. Since the goal is to estimate the disease control rate, the trial is not stopped for futility after the first stage if the disease control rate is promising. The new design can be generated using easy-to-use software that is available at http://cancer.unc.edu/biostatistics/program/ivanova/.

DOI10.4310/SII.2016.v9.n1.a9
Alternate JournalStat Interface
Original PublicationTwo-stage design for phase II oncology trials with relaxed futility stopping.
PubMed ID29805727
PubMed Central IDPMC5969536
Grant ListP01 CA142538 / CA / NCI NIH HHS / United States
R01 CA120082 / CA / NCI NIH HHS / United States
Project: