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Open Access Publications from the University of California

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

  • Author(s): Stamelou, Maria
  • Schöpe, Jakob
  • Wagenpfeil, Stefan
  • Del Ser, Teodoro
  • Bang, Jee
  • Lobach, Iryna Y
  • Luong, Phi
  • Respondek, Gesine
  • Oertel, Wolfgang H
  • Boxer, AdamL
  • Höglinger, Günter U
  • AL-108-231 Investigators, Tauros Investigators, and MDS-Endorsed PSP Study Group
  • et al.

Published Web Location

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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