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Verfasst von:Jensen, Katrin [VerfasserIn]   i
 Kieser, Meinhard [VerfasserIn]   i
Titel:Blinded sample size recalculation in multicentre trials with normally distributed outcome
Verf.angabe:Katrin Jensen and Meinhard Kieser (Institute of Medical Biometry and Informatics, Ruprecht-Karls University Heidelberg)
E-Jahr:2010
Jahr:17 June 2010
Umfang:23 S.
Fussnoten:Gesehen am 24.08.2023
Titel Quelle:Enthalten in: Biometrical journal
Ort Quelle:Berlin : Wiley-VCH, 1977
Jahr Quelle:2010
Band/Heft Quelle:52(2010), 3 vom: Juni, Seite 377-399
ISSN Quelle:1521-4036
Abstract:The internal pilot study design enables to estimate nuisance parameters required for sample size calculation on the basis of data accumulated in an ongoing trial. By this, misspecifications made when determining the sample size in the planning phase can be corrected employing updated knowledge. According to regulatory guidelines, blindness of all personnel involved in the trial has to be preserved and the specified type I error rate has to be controlled when the internal pilot study design is applied. Especially in the late phase of drug development, most clinical studies are run in more than one centre. In these multicentre trials, one may have to deal with an unequal distribution of the patient numbers among the centres. Depending on the type of the analysis (weighted or unweighted), unequal centre sample sizes may lead to a substantial loss of power. Like the variance, the magnitude of imbalance is difficult to predict in the planning phase. We propose a blinded sample size recalculation procedure for the internal pilot study design in multicentre trials with normally distributed outcome and two balanced treatment groups that are analysed applying the weighted or the unweighted approach. The method addresses both uncertainty with respect to the variance of the endpoint and the extent of disparity of the centre sample sizes. The actual type I error rate as well as the expected power and sample size of the procedure is investigated in simulation studies. For the weighted analysis as well as for the unweighted analysis, the maximal type I error rate was not or only minimally exceeded. Furthermore, application of the proposed procedure led to an expected power that achieves the specified value in many cases and is throughout very close to it.
DOI:doi:10.1002/bimj.200900114
URL:Bitte beachten Sie: Dies ist ein Bibliographieeintrag. Ein Volltextzugriff für Mitglieder der Universität besteht hier nur, falls für die entsprechende Zeitschrift/den entsprechenden Sammelband ein Abonnement besteht oder es sich um einen OpenAccess-Titel handelt.

Volltext: https://doi.org/10.1002/bimj.200900114
 Volltext: https://onlinelibrary.wiley.com/doi/abs/10.1002/bimj.200900114
 DOI: https://doi.org/10.1002/bimj.200900114
Datenträger:Online-Ressource
Sprache:eng
Sach-SW:Internal pilot study
 Multicentre clinical trial
 Nuisance parameter
 Power
 Sample size recalculation
 Type I error
 Variance estimate
K10plus-PPN:1857898702
Verknüpfungen:→ Zeitschrift

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