Single-arm Phase II cancer survival trial designs

Jianrong Wu

Research output: Contribution to journalArticlepeer-review

4 Scopus citations

Abstract

The current practice for designing single-arm Phase II trials with time-to-event endpoints is limited to using either a maximum likelihood estimate test under the exponential model or a naive approach based on dichotomizing the event time at a landmark time point. A trial designed under the exponential model may not be reliable, and the naive approach is inefficient. The modified one-sample log-rank test statistic proposed in this article fills the void. In general, the proposed test can be used to design single-arm Phase II survival trials under any parametric survival distribution. Simulation results showed that it preserves type I error well and provides adequate power for Phase II cancer trial designs with time-to-event endpoints.

Original languageEnglish
Pages (from-to)644-656
Number of pages13
JournalJournal of Biopharmaceutical Statistics
Volume26
Issue number4
DOIs
StatePublished - Jul 3 2016

Bibliographical note

Funding Information:
This work was supported in part by the National Cancer Institute (NCI) support grant P30CA021765 and the American Lebanese Syrian Associated Charities (ALSAC).

Publisher Copyright:
© 2016 Taylor & Francis.

Keywords

  • Clinical trial design
  • one-sample log-rank test
  • sample size
  • single-arm Phase II
  • survival analysis
  • time-to-event

ASJC Scopus subject areas

  • Statistics and Probability
  • Pharmacology
  • Pharmacology (medical)

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