A stepped wedge design for testing an effect of intranasal insulin on cognitive development of children with Phelan-McDermid syndrome: A comparison of different designs

E.R. van den Heuvel, R.J. Zwanenburg, C.M.A. Van Ravenswaaij-Arts

Research output: Contribution to journalReview articleAcademicpeer-review

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Abstract

This paper compares the power of the parallel group design, the matched-pairs design, and several options for the stepped wedge and delayed start designs for testing a possible effect of intranasal insulin with respect to placebo on developmental growth of children with a rare disorder like Phelan-McDermid syndrome. A subject-specific linear mixed effects model for the primary outcome developmental age in a longitudinal setting with five time points was assumed. Monte Carlo simulation studies with small sample sizes were applied since the rare disorder prohibits large trials. The stepped wedge designs, which were initially preferred for ethical reasons, appear to be competitive in power to other designs and were in some settings even the best. The assumed statistical model also demonstrates that all of the designs can be viewed as a stepped wedge or delayed treatment design. Our results show that the stepped wedge design is an appropriate alternative for randomized controlled trials on developmental growth with small numbers of participants under the formulated statistical conditions.

Original languageEnglish
Pages (from-to)766-775
Number of pages10
JournalStatistical Methods in Medical Research
Volume26
Issue number2
DOIs
Publication statusPublished - 1 Apr 2017

Keywords

  • delayed start design
  • Developmental growth
  • Phelan-McDermid syndrome
  • random coefficient model
  • small trial designs
  • stepped wedge design

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