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Lookup NU author(s): Professor David BurnORCiD, Laura Morris
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© 2016 International Parkinson and Movement Disorder Society. 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.
Author(s): Stamelou M, Schope J, Wagenpfeil S, Del Ser T, Bang J, Lobach IY, Luong P, Respondek G, Oertel WH, Boxer A, Hoglinger GU, Williams D, Lafontaine AL, Marras C, Jog M, Panisset M, Lang A, Parker L, Stewart AJ, Corvol J-C, Azulay J-P, Couratier P, Mollenhauer B, Lorenzl S, Ludolph A, Benecke R, Hoglinger G, Lipp A, Reichmann H, Woitalla D, Chan D, Zermansky A, Burn D, Lees A, Boxer A, Miller BL, Lobach IV, Roberson E, Honig L, Zamrini E, Pahwa R, Bordelon Y, Driver-Dunkley E, Lessig S, Lew M, Womack K, Boeve B, Ferrara J, Hillis A, Kaufer D, Kumar R, Xie T, Gunzler S, Zesiewicz T, Dayalu P, Golbe L, Grossman M, Jankovic J, McGinnis S, Santiago A, Tuite P, Isaacson S, Leegwater-Kim J, Litvan I, Grossman M, Knopman DS, Schneider LS, Doody RS, Golbe L, Koestler M, Jack CR, Van Deerlin V, Randolph C, Gozes I, Whitaker S, Hirman J, Gold M, Morimoto BH, Gomez JC, Tijero B, Berganzo K, Garcia de Yebenes J, Lopez Sendon JL, Garcia G, Tolosa E, Buongiorno MT, Bargallo N, Burguera JA, Martinez I, Ruiz-Martinez J, Narrativel I, Vivancos F, Ybot I, Aguilar M, Quilez P, Boada M, Lafuente A, Hernandez I, Lopez-Lozano JJ, Mata M, Kupsch A, Lipp A, Ebersbach G, Schmidt T, Hahn K, Hoglinger G, Hollerhage M, Reichmann H, Wolz M, Schneider C, Klingelhofer L, Berg D, Maetzler W, Srulijes KK, Ludolph A, Kassubek J, Steiger M, Tyler K, Burn DJ, Morris L, Lees A, Ling H, Hauser R, McClain T, Truong D, Jenkins S, Litvan I, Houghton D, Ferrara J, Bordelon Y, Gratiano A, Golbe L, Mark M, Uitti R, Ven Gerpen J, Bhatia K, Bordelon YM, Colosimo C, Dodel R, Josephs KA, Morris H, Mueller U, Paviour D, Schellenberg G, Steele J, van Swieten JC, Whitwell J
Publication type: Article
Publication status: Published
Journal: Movement Disorders
Year: 2016
Volume: 31
Issue: 5
Pages: 742-747
Online publication date: 07/03/2016
Acceptance date: 24/01/2016
ISSN (print): 0885-3185
ISSN (electronic): 1531-8257
Publisher: John Wiley and Sons Inc.
URL: http://doi.org/10.1002/mds.26580
DOI: 10.1002/mds.26580
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