1. Phase II trial design with growth modulation index as the primary endpoint.
- Author
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Wu, Jianrong, Chen, Li, Wei, Jing, Weiss, Heidi, Miller, Rachel W., and Villano, John L.
- Subjects
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CANCER relapse , *LOG-linear models , *TEST scoring , *CLINICAL trials , *SOUND design - Abstract
Molecularly targeted, genomic‐driven, and immunotherapy‐based clinical trials continue to be advanced for the treatment of relapse or refractory cancer patients, where the growth modulation index (GMI) is often considered a primary endpoint of treatment efficacy. However, there little literature is available that considers the trial design with GMI as the primary endpoint. In this article, we derived a sample size formula for the score test under a log‐linear model of the GMI. Study designs using the derived sample size formula are illustrated under a bivariate exponential model, the Weibull frailty model, and the generalized treatment effect size. The proposed designs provide sound statistical methods for a single‐arm phase II trial with GMI as the primary endpoint. [ABSTRACT FROM AUTHOR]
- Published
- 2019
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