Phase II trial design with growth modulation index as the primary endpoint

Jianrong Wu, Li Chen, Jing Wei, Heidi Weiss, Rachel W. Miller, John L. Villano

Research output: Contribution to journalArticlepeer-review

3 Scopus citations

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.

Original languageEnglish
Pages (from-to)212-222
Number of pages11
JournalPharmaceutical Statistics
Volume18
Issue number2
DOIs
StatePublished - Mar 1 2019

Bibliographical note

Publisher Copyright:
© 2018 John Wiley & Sons, Ltd.

Keywords

  • accelerated failure time model
  • clinical trial design
  • growth modulation Index
  • paired time-to-progression
  • sample size

ASJC Scopus subject areas

  • Statistics and Probability
  • Pharmacology
  • Pharmacology (medical)

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