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

Lookup NU author(s): Professor David Burn, 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.

Publication metadata

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.


DOI: 10.1002/mds.26580


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