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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
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.
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3rd International Clinical Trials Methodology Conference