Using historical lesion volume data in the design of a new phase II clinical trial in acute stroke

John Whitehead, Kim Bolland, Elsa Valdès-Márquez, Anela Lihic, Myzoon Ali, Kennedy J. Lees

    Research output: Contribution to journalArticle

    Abstract

    Clinical research into the treatment of acute stroke is complicated, is costly, and has often been unsuccessful. Developments in imaging technology based on computed tomography and magnetic resonance imaging scans offer opportunities for screening experimental therapies during phase II testing so as to deliver only the most promising interventions to phase III. We discuss the design and the appropriate sample size for phase II studies in stroke based on lesion volume. Determination of the relation between analyses of lesion volumes and of neurologic outcomes is illustrated using data from placebo trial patients from the Virtual International Stroke Trials Archive. The size of an effect on lesion volume that would lead to a clinically relevant treatment effect in terms of a measure, such as modified Rankin score (mRS), is found. The sample size to detect that magnitude of effect on lesion volume is then calculated. Simulation is used to evaluate different criteria for proceeding from phase II to phase III.

    Original languageEnglish
    Pages (from-to)1347-1352
    Number of pages6
    JournalStroke
    Volume40
    Issue number4
    Early online date19 Feb 2009
    DOIs
    Publication statusPublished - Apr 2009

    Keywords

    • acute stroke
    • VISTA
    • clinical trials

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