Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2012 Feb 20;30(6):667-71.
doi: 10.1200/JCO.2011.38.5732. Epub 2012 Jan 23.

Design issues in randomized phase II/III trials

Edward L Korn  1 , Boris Freidlin, Jeffrey S Abrams, Susan Halabi
Affiliations

Design issues in randomized phase II/III trials

Edward L Korn et al. J Clin Oncol. .

Abstract

Phase II trials are used to show sufficient preliminary activity of a new treatment (in single-arm designs or randomized screening designs) or to select among treatments with demonstrated activity (in randomized selection designs). The treatments prioritized in a phase II trial are then tested definitively against a control treatment in a randomized phase III trial. Randomized phase II/III trials use an adaptive trial design that combines these two types of trials in one, with potential gains in time and reduced numbers of patients required to be treated. Two key considerations in designing a phase II/III trial are whether to suspend accrual while the phase II data mature and the choice of phase II target treatment effect. We discuss these phase II/III design parameters, give examples of phase II/III trials, and provide recommendations concerning efficient phase II/III trial designs.

PubMed Disclaimer

Conflict of interest statement

Authors' disclosures of potential conflicts of interest and author contributions are found at the end of this article.

References

    1. Thall PF, Simon R, Ellenberg SS. Two-stage selection and testing designs for comparative clinical trials. Biometrika. 1988;75:303–310.
    1. Schaid DJ, Wieand S, Therneau TM. Optimal two-stage screening designs for survival comparisons. Biometrika. 1990;77:507–513.
    1. Scher HI, Heller G. Picking the winners in a sea of plenty. Clin Cancer Res. 2002;8:400–404. - PubMed
    1. Bretz F, Schmidli H, König F, et al. Confirmatory seamless phase II/III clinical trials with hypothesis selection at interim: General concepts. Biom J. 2006;48:623–634. - PubMed
    1. Maca J, Bhattacharya S, Dragalin V, et al. Adaptive seamless phase II/III designs: Background, operational aspects, and examples. Drug Inf J. 2006;40:463–473.

MeSH terms