A Three-Outcome Design for Phase II Clinical Trials

by: Daniel J Sargent, Victor Chan, Richard M Goldberg

Controlled Clinical Trials, Vol. 22, No. 2. (February 2001), pp. 117-125.

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摘要

The goal of a phase II trial is to make a preliminary determination regarding the activity and tolerability of a new treatment and thus to determine whether the treatment warrants further study in the phase III setting. Phase II clinical trials are typically designed in the hypothesis testing framework with two possible outcomes, either reject the null hypothesis H0 or reject the alternative hypothesis Ha, based on the observed activity level. However, in cases where the observed activity is "borderline," the decision regarding the future of the agent is not as clear as the prespecified hypothesis test would indicate. In this paper we propose an alternative design that allows for three outcomes: reject H0, reject Ha, or reject neither. We describe the theoretical properties of this design and illustrate it with several examples. We focus on the clinical implications of the three-outcome design. Control Clin Trials 2001;22:117-125

@article{citeulike:2465059, abstract = {The goal of a phase II trial is to make a preliminary determination regarding the activity and tolerability of a new treatment and thus to determine whether the treatment warrants further study in the phase III setting. Phase II clinical trials are typically designed in the hypothesis testing framework with two possible outcomes, either reject the null hypothesis H0 or reject the alternative hypothesis Ha, based on the observed activity level. However, in cases where the observed activity is "borderline," the decision regarding the future of the agent is not as clear as the prespecified hypothesis test would indicate. In this paper we propose an alternative design that allows for three outcomes: reject H0, reject Ha, or reject neither. We describe the theoretical properties of this design and illustrate it with several examples. We focus on the clinical implications of the three-outcome design. Control Clin Trials 2001;22:117-125}, author = {Sargent, Daniel J. and Chan, Victor and Goldberg, Richard M. }, citeulike-article-id = {2465059}, doi = {http://dx.doi.org/10.1016/S0197-2456(00)00115-X}, journal = {Controlled Clinical Trials}, keywords = {clinical\_trial, phase\_ii, three\_outcome\_design}, month = {February}, number = {2}, pages = {117--125}, posted-at = {2008-03-04 15:00:13}, priority = {2}, title = {A Three-Outcome Design for Phase II Clinical Trials}, url = {http://dx.doi.org/10.1016/S0197-2456(00)00115-X}, volume = {22}, year = {2001} }

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TY - JOUR ID - citeulike:2465059 L3 - citeulike-article-id:2465059 N2 - The goal of a phase II trial is to make a preliminary determination regarding the activity and tolerability of a new treatment and thus to determine whether the treatment warrants further study in the phase III setting. Phase II clinical trials are typically designed in the hypothesis testing framework with two possible outcomes, either reject the null hypothesis H0 or reject the alternative hypothesis Ha, based on the observed activity level. However, in cases where the observed activity is "borderline," the decision regarding the future of the agent is not as clear as the prespecified hypothesis test would indicate. In this paper we propose an alternative design that allows for three outcomes: reject H0, reject Ha, or reject neither. We describe the theoretical properties of this design and illustrate it with several examples. We focus on the clinical implications of the three-outcome design. Control Clin Trials 2001;22:117-125 IS - 2 JF - Controlled Clinical Trials EP - 125 TI - A Three-Outcome Design for Phase II Clinical Trials VL - 22 SP - 117 KW - clinical_trial KW - phase_ii KW - three_outcome_design AU - Sargent, Daniel AU - Chan, Victor AU - Goldberg, Richard PY - 2001/02// UR - http://dx.doi.org/10.1016/S0197-2456(00)00115-X ER -

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