This paper discusses results reported in The Lancet Neurology, by the European Friedreich’s Ataxia Consortium for Translational Studies (EFACTS). EFACTS presented their data from a 2 year natural history study of individuals with Friedreich’s ataxia. The data show the progression of various neurological outcome measures over the course of 2 years. In this paper, the authors explain that EFACTS concluded that their projections suggest that for clinical trials in Friedreich’s ataxia using current measures to demonstrate that a compound is effective, they are likely to need to be carried out over 2 years, and even then will require more than 180 participants. They explain that the US natural history study has shown similar results, which suggests that most trials to date are too short and too small to show the effect of a potential drug. They suggest that future trials should be both longer and larger, or that perhaps patients could be stratified so that more uniform populations of patients with more rapid progression could be used, so as to reduce the time and length of trial needed to show a positive effect of a potential therapeutic.
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