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Flexible Phase I Clinical Trials: Allowing for Nonbinary Toxicity Response and Removal of Other Common Limitations.

Publication ,  Journal Article
Potthoff, RF; George, SL
Published in: Stat Biopharm Res
August 1, 2009

Phase I clinical trials are often subject to severe limitations. The most important one is that they typically allow only for binary response-toxic (1) or nontoxic (0)-rather than a range of responses from 0 to 1. They also may not allow a new patient to be treated until results for all previous patients are available. They may assign patients to doses in groups of two or more, rather than individually. They may require the selected dose to be one of a few prespecified doses. The flexible method proposed here addresses these four limitations. It adopts a quasi-Bayesian approach incorporating a logistic dose-response model with two parameters for the mean response. The response at any dose follows a beta distribution, which entails a third parameter. The choice of dose for a patient is based on a utility function that reflects the latest estimates of toxicity and of the variance of the estimate of the maximum tolerated dose (MTD). Simulations show that the method works well, and that a nonbinary toxicity measure leads to a far more accurate MTD estimate than does a binary one.

Duke Scholars

Published In

Stat Biopharm Res

DOI

ISSN

1946-6315

Publication Date

August 1, 2009

Volume

1

Issue

3

Start / End Page

213 / 228

Location

United States

Related Subject Headings

  • 4905 Statistics
  • 0104 Statistics
 

Citation

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Potthoff, R. F., & George, S. L. (2009). Flexible Phase I Clinical Trials: Allowing for Nonbinary Toxicity Response and Removal of Other Common Limitations. Stat Biopharm Res, 1(3), 213–228. https://doi.org/10.1198/sbr.2009.0014
Potthoff, Richard F., and Stephen L. George. “Flexible Phase I Clinical Trials: Allowing for Nonbinary Toxicity Response and Removal of Other Common Limitations.Stat Biopharm Res 1, no. 3 (August 1, 2009): 213–28. https://doi.org/10.1198/sbr.2009.0014.
Potthoff, Richard F., and Stephen L. George. “Flexible Phase I Clinical Trials: Allowing for Nonbinary Toxicity Response and Removal of Other Common Limitations.Stat Biopharm Res, vol. 1, no. 3, Aug. 2009, pp. 213–28. Pubmed, doi:10.1198/sbr.2009.0014.
Journal cover image

Published In

Stat Biopharm Res

DOI

ISSN

1946-6315

Publication Date

August 1, 2009

Volume

1

Issue

3

Start / End Page

213 / 228

Location

United States

Related Subject Headings

  • 4905 Statistics
  • 0104 Statistics