Sample size determination in group-sequential clinical trials with two co-primary endpoints

Koko Asakura, Toshimitsu Hamasaki, Tomoyuki Sugimoto, Kenichi Hayashi, Scott R. Evans, Takashi Sozu

Research output: Contribution to journalArticle

12 Citations (Scopus)

Abstract

We discuss sample size determination in group-sequential designs with two endpoints as co-primary. We derive the power and sample size within two decision-making frameworks. One is to claim the test intervention's benefit relative to control when superiority is achieved for the two endpoints at the same interim timepoint of the trial. The other is when superiority is achieved for the two endpoints at any interim timepoint, not necessarily simultaneously. We evaluate the behaviors of sample size and power with varying design elements and provide a real example to illustrate the proposed sample size methods. In addition, we discuss sample size recalculation based on observed data and evaluate the impact on the power and Type I error rate.

Original languageEnglish
Pages (from-to)2897-2913
Number of pages17
JournalStatistics in Medicine
Volume33
Issue number17
DOIs
Publication statusPublished - 2014 Jul 30
Externally publishedYes

Fingerprint

Group Sequential
Sample Size Determination
Clinical Trials
Sample Size
Group Sequential Design
Type I Error Rate
Evaluate
Decision Making
Power (Psychology)

Keywords

  • Average sample number
  • Co-primary endpoints
  • Conditional power
  • Cui-Hung-Wang statistics
  • Group-sequential methods
  • Maximum sample size
  • Sample size recalculation
  • Type I error

ASJC Scopus subject areas

  • Epidemiology
  • Statistics and Probability
  • Medicine(all)

Cite this

Asakura, K., Hamasaki, T., Sugimoto, T., Hayashi, K., Evans, S. R., & Sozu, T. (2014). Sample size determination in group-sequential clinical trials with two co-primary endpoints. Statistics in Medicine, 33(17), 2897-2913. https://doi.org/10.1002/sim.6154

Sample size determination in group-sequential clinical trials with two co-primary endpoints. / Asakura, Koko; Hamasaki, Toshimitsu; Sugimoto, Tomoyuki; Hayashi, Kenichi; Evans, Scott R.; Sozu, Takashi.

In: Statistics in Medicine, Vol. 33, No. 17, 30.07.2014, p. 2897-2913.

Research output: Contribution to journalArticle

Asakura, K, Hamasaki, T, Sugimoto, T, Hayashi, K, Evans, SR & Sozu, T 2014, 'Sample size determination in group-sequential clinical trials with two co-primary endpoints', Statistics in Medicine, vol. 33, no. 17, pp. 2897-2913. https://doi.org/10.1002/sim.6154
Asakura K, Hamasaki T, Sugimoto T, Hayashi K, Evans SR, Sozu T. Sample size determination in group-sequential clinical trials with two co-primary endpoints. Statistics in Medicine. 2014 Jul 30;33(17):2897-2913. https://doi.org/10.1002/sim.6154
Asakura, Koko ; Hamasaki, Toshimitsu ; Sugimoto, Tomoyuki ; Hayashi, Kenichi ; Evans, Scott R. ; Sozu, Takashi. / Sample size determination in group-sequential clinical trials with two co-primary endpoints. In: Statistics in Medicine. 2014 ; Vol. 33, No. 17. pp. 2897-2913.
@article{ae4c49bc9eee40eeae23b67a24a8428d,
title = "Sample size determination in group-sequential clinical trials with two co-primary endpoints",
abstract = "We discuss sample size determination in group-sequential designs with two endpoints as co-primary. We derive the power and sample size within two decision-making frameworks. One is to claim the test intervention's benefit relative to control when superiority is achieved for the two endpoints at the same interim timepoint of the trial. The other is when superiority is achieved for the two endpoints at any interim timepoint, not necessarily simultaneously. We evaluate the behaviors of sample size and power with varying design elements and provide a real example to illustrate the proposed sample size methods. In addition, we discuss sample size recalculation based on observed data and evaluate the impact on the power and Type I error rate.",
keywords = "Average sample number, Co-primary endpoints, Conditional power, Cui-Hung-Wang statistics, Group-sequential methods, Maximum sample size, Sample size recalculation, Type I error",
author = "Koko Asakura and Toshimitsu Hamasaki and Tomoyuki Sugimoto and Kenichi Hayashi and Evans, {Scott R.} and Takashi Sozu",
year = "2014",
month = "7",
day = "30",
doi = "10.1002/sim.6154",
language = "English",
volume = "33",
pages = "2897--2913",
journal = "Statistics in Medicine",
issn = "0277-6715",
publisher = "John Wiley and Sons Ltd",
number = "17",

}

TY - JOUR

T1 - Sample size determination in group-sequential clinical trials with two co-primary endpoints

AU - Asakura, Koko

AU - Hamasaki, Toshimitsu

AU - Sugimoto, Tomoyuki

AU - Hayashi, Kenichi

AU - Evans, Scott R.

AU - Sozu, Takashi

PY - 2014/7/30

Y1 - 2014/7/30

N2 - We discuss sample size determination in group-sequential designs with two endpoints as co-primary. We derive the power and sample size within two decision-making frameworks. One is to claim the test intervention's benefit relative to control when superiority is achieved for the two endpoints at the same interim timepoint of the trial. The other is when superiority is achieved for the two endpoints at any interim timepoint, not necessarily simultaneously. We evaluate the behaviors of sample size and power with varying design elements and provide a real example to illustrate the proposed sample size methods. In addition, we discuss sample size recalculation based on observed data and evaluate the impact on the power and Type I error rate.

AB - We discuss sample size determination in group-sequential designs with two endpoints as co-primary. We derive the power and sample size within two decision-making frameworks. One is to claim the test intervention's benefit relative to control when superiority is achieved for the two endpoints at the same interim timepoint of the trial. The other is when superiority is achieved for the two endpoints at any interim timepoint, not necessarily simultaneously. We evaluate the behaviors of sample size and power with varying design elements and provide a real example to illustrate the proposed sample size methods. In addition, we discuss sample size recalculation based on observed data and evaluate the impact on the power and Type I error rate.

KW - Average sample number

KW - Co-primary endpoints

KW - Conditional power

KW - Cui-Hung-Wang statistics

KW - Group-sequential methods

KW - Maximum sample size

KW - Sample size recalculation

KW - Type I error

UR - http://www.scopus.com/inward/record.url?scp=84903817449&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84903817449&partnerID=8YFLogxK

U2 - 10.1002/sim.6154

DO - 10.1002/sim.6154

M3 - Article

VL - 33

SP - 2897

EP - 2913

JO - Statistics in Medicine

JF - Statistics in Medicine

SN - 0277-6715

IS - 17

ER -