title: Alternative trial design in amyotrophic lateral sclerosis saves time and patients.
abstract: A sequential trial design is an alternative for the classical trial design with a fixed sample size, that permits stopping a trial as soon as enough evidence for a treatment effect, or a lack thereof, is obtained. This study aimed to determine the difference in efficiency of time and patient number between a classical trial design and a sequential trial design. In this study we re-analysed a previously published classically designed clinical trial according to a sequential trial design. We subsequently determined the difference in total running time and patient number. We found that the sequential analysis offered a gain in time of 38%. We conclude that the sequential trial design may in certain situations be superior to the classical design.
authors.affiliation: The Department of Neurology, University Medical Centre-Utrecht, Utrecht, The Netherlands. g.j.groeneveld@umcutrecht.nl
authors.forename: Geert Jan
authors.initials: GJ
authors.lastname: Groeneveld
authors.forename: Michael
authors.initials: M
authors.lastname: Graf
authors.forename: Ingeborg
authors.initials: I
authors.lastname: van der Tweel
authors.forename: Leonard H
authors.initials: LH
authors.lastname: van den Berg
authors.forename: Albert C
authors.initials: AC
authors.lastname: Ludolph
mesh_terms: D000690:Amyotrophic Lateral Sclerosis / Q000191:economics* / Q000628:therapy*; D002986:Clinical Trials as Topic / Q000191:economics* / Q000379:methods; D016001:Confidence Intervals; D004311:Double-Blind Method; D006801:Humans; D016017:Odds Ratio; D012107:Research Design; D012189:Retrospective Studies; D018401:Sample Size; D016019:Survival Analysis; D013997:Time Factors; D014810:Vitamin E / Q000627:therapeutic use*
chemical_list: D014810:Vitamin E