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. 2010 Mar 15;16(6):1764-9.
doi: 10.1158/1078-0432.CCR-09-3287. Epub 2010 Mar 9.

The design of phase II clinical trials testing cancer therapeutics: consensus recommendations from the clinical trial design task force of the national cancer institute investigational drug steering committee

Lesley Seymour  1 , S Percy Ivy, Daniel Sargent, David Spriggs, Laurence Baker, Larry Rubinstein, Mark J Ratain, Michael Le Blanc, David Stewart, John Crowley, Susan Groshen, Jeffrey S Humphrey, Pamela West, Donald Berry
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The design of phase II clinical trials testing cancer therapeutics: consensus recommendations from the clinical trial design task force of the national cancer institute investigational drug steering committee

Lesley Seymour et al. Clin Cancer Res. .

Abstract

The optimal design of phase II studies continues to be the subject of vigorous debate, especially studies of newer molecularly targeted agents. The observations that many new therapeutics "fail" in definitive phase III studies, coupled with the numbers of new agents to be tested as well as the increasing costs and complexity of clinical trials, further emphasize the critical importance of robust and efficient phase II design. The Clinical Trial Design Task Force (CTD-TF) of the National Cancer Institute (NCI) Investigational Drug Steering Committee (IDSC) has published a series of discussion papers on phase II trial design in Clinical Cancer Research. The IDSC has developed formal recommendations about aspects of phase II trial design that are the subject of frequent debate, such as endpoints (response versus progression-free survival), randomization (single-arm designs versus randomization), inclusion of biomarkers, biomarker-based patient enrichment strategies, and statistical design (e.g., two-stage designs versus multiple-group adaptive designs). Although these recommendations in general encourage the use of progression-free survival as the primary endpoint, randomization, inclusion of biomarkers, and incorporation of newer designs, we acknowledge that objective response as an endpoint and single-arm designs remain relevant in certain situations. The design of any clinical trial should always be carefully evaluated and justified based on characteristic specific to the situation.

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Figures

Figure 1
Figure 1. Process for Development of Recommendations
IDSC – Investigational Drug Steering Committee
Figure 2
Figure 2
Process/Flow or Approaches for determination of Phase 2 trial design recommendations. RCT – randomized controlled trial, 20 – secondary, PRO – patient related outcomes, PFS = progression-free survival.
Figure 3
Figure 3
Types of Phase II Studies
See this image and copyright information in PMC

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