@article{0321408254984d8895a17522dffde2c5,
title = "Power calculations and placebo effect for future clinical trials in progressive supranuclear palsy",
abstract = "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.",
keywords = "Clinical trials, Placebo effect, Power calculation, Progressive supranuclear palsy, Rate of progression",
author = "{Tau Restoration on PSP (TAUROS) Investigators} and {The MDS-Endorsed PSP Study Group} and Maria Stamelou and Jakob Sch{\"o}pe and Stefan Wagenpfeil and {Del Ser}, Teodoro and Jee Bang and Lobach, {Iryna Y.} and Phi Luong and Gesine Respondek and Oertel, {Wolfgang H.} and Adaml Boxer and H{\"o}glinger, {G{\"u}nter U.} and David Williams and Lafontaine, {Anne Louise} and Connie Marras and Mandar Jog and Michael Panisset and Anthony Lang and Lesley Parker and Stewart, {Alistair J.} and Corvol, {Jean Christophe} and Azulay, {Jean Philippe} and Philippe Couratier and Brit Mollenhauer and Stefan Lorenzl and A. Ludolph and Reiner Benecke and G. Hoglinger and A. Lipp and H. Reichmann and Dirk Woitalla and Dennis Chan and Adam Zermansky and Burn, {David J.} and A. Lees and Adam Boxer and Miller, {Bruce L.} and Lobach, {Iryna V.} and Erik Roberson and Lawrence Honig and Edward Zamrini and Rajesh Pahwa and Y. Bordelon and Erika Driver-Dunkley and Stephanie Lessig and Mark Lew and Kyle Womack and Brad Boeve and J. Ferrara and Argyle Hillis and Daniel Kaufer",
note = "Publisher Copyright: {\textcopyright} 2016 International Parkinson and Movement Disorder Society.",
year = "2016",
month = may,
day = "1",
doi = "10.1002/mds.26580",
language = "English",
volume = "31",
pages = "742--747",
journal = "Movement Disorders",
issn = "0885-3185",
number = "5",
}