Blog
About

9
views
0
recommends
+1 Recommend
0 collections
    0
    shares
      • Record: found
      • Abstract: found
      • Article: found
      Is Open Access

      Two-arm randomised futility trials: PD-stat - a futility trial of a potential neuroprotective treatment in people with Parkinson's disease

      , 1 , 1 , 1 , 2 , 3 , 4 , 1

      Trials

      BioMed Central

      3rd International Clinical Trials Methodology Conference

      16-17 November 2015

      Read this article at

      Bookmark
          There is no author summary for this article yet. Authors can add summaries to their articles on ScienceOpen to make them more accessible to a non-specialist audience.

          Abstract

          Futility trials are efficient, early phase studies designed to eliminate potential interventions or treatments before moving into large and expensive, definitive phase III trials. In a futility trial, the null and alternative hypotheses are reversed in comparison with the usual superiority trial and are one-sided. The null hypothesis is that the intervention reaches or exceeds the required level of benefit. Initially used in oncology, where such studies are usually simply referred to as phase II trials and have relatively short follow-up periods, there has been increasing interest in the use of the futility trial design in other clinical areas, in particular in neurological diseases. Typically, such futility studies have had a single arm and have tested whether the new treatment exceeds a pre-defined futility threshold, set as the minimum response worthwhile to justify moving to a definitive trial. However, in neurological diseases, such as Parkinson's disease, the lack of a concurrent control group has led to criticism of the subsequent findings. In an attempt to overcome such issues, it is possible to use a randomised two-arm design, whilst still testing for futility. PD-STAT, funded by the Cure Parkinson's Trust (CPT) and JP Moulton Charitable Foundation as part of the CPT's Linked Clinical Trials Initiative, is the first UK study to utilise this design in Parkinson's disease. We will describe the design characteristics and planned analysis of this trial and outline some of the advantages and challenges associated with futility trials.

          Related collections

          Author and article information

          Conference
          Trials
          Trials
          Trials
          BioMed Central
          1745-6215
          2015
          16 November 2015
          : 16
          : Suppl 2
          : P236
          Affiliations
          [1 ]Plymouth University, Plymouth, UK
          [2 ]University of St Andrews, St Andrews, UK
          [3 ]University of Exeter, Exeter, UK
          [4 ]Medical University of South Carolina, South Carolina, USA
          Article
          1745-6215-16-S2-P236
          10.1186/1745-6215-16-S2-P236
          4660100
          Copyright © 2015 Creanor et al.

          This is an Open Access article distributed under the terms of the Creative Commons Attribution License ( http://creativecommons.org/licenses/by/4.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. The Creative Commons Public Domain Dedication waiver ( http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.

          3rd International Clinical Trials Methodology Conference
          Glasgow, UK
          16-17 November 2015
          Categories
          Poster Presentation

          Medicine

          Comments

          Comment on this article