Power calculations and placebo effect for future clinical trials in progressive supranuclear palsy.

Mov Disord 2016 05 7;31(5):742-7. Epub 2016 Mar 7.

Department of Neurology, Philipps University, Marburg, Germany.

Background: Two recent randomized, placebo-controlled trials of putative disease-modifying agents (davunetide, tideglusib) in progressive supranuclear palsy (PSP) failed to show efficacy, but generated data relevant for future trials.

Methods: We provide sample size calculations based on data collected in 187 PSP patients assigned to placebo in these trials. A placebo effect was calculated.

Results: The total PSP-Rating Scale required the least number of patients per group (N = 51) to detect a 50% change in the 1-year progression and 39 when including patients with ≤ 5 years disease duration. The Schwab and England Activities of Daily Living required 70 patients per group and was highly correlated with the PSP-Rating Scale. A placebo effect was not detected in these scales.

Conclusions: We propose the 1-year PSP-Rating Scale score change as the single primary readout in clinical neuroprotective or disease-modifying trials. The Schwab and England Activities of Daily Living could be used as a secondary outcome. © 2016 International Parkinson and Movement Disorder Society.

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http://dx.doi.org/10.1002/mds.26580DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5289149PMC
May 2016
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