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

Maria Stamelou, Jakob Schöpe, Stefan Wagenpfeil, Teodoro Del Ser, Jee Bang, Iryna Y. Lobach, Phi Luong, Gesine Respondek, Wolfgang H. Oertel, Adaml Boxer, Günter U. Höglinger, David Williams, Anne Louise Lafontaine, Connie Marras, Mandar Jog, Michael Panisset, Anthony Lang, Lesley Parker, Alistair J. Stewart, Jean Christophe CorvolJean Philippe Azulay, Philippe Couratier, Brit Mollenhauer, Stefan Lorenzl, A. Ludolph, Reiner Benecke, G. Hoglinger, A. Lipp, H. Reichmann, Dirk Woitalla, Dennis Chan, Adam Zermansky, David Burn, A. Lees, Adam Boxer, Bruce L. Miller, Iryna V. Lobach, Erik Roberson, Lawrence Honig, Edward Zamrini, Rajesh Pahwa, Y. Bordelon, Erika Driver-Dunkley, Stephanie Lessig, Mark Lew, Kyle Womack, Brad Boeve, J. Ferrara, Argyle Hillis, Daniel Kaufer, Rajeev Kumar, Tao Xie, Steven Gunzler, Theresa Zesiewicz, Praveen Dayalu, L. Golbe, Murray Grossman, Joseph Jankovic, Scott McGinnis, Anthony Santiago, Paul Tuite, Stuart Isaacson, Julie Leegwater-Kim, I. Litvan, Murray Grossman, David S. Knopman, Lon S. Schneider, Rachelle S. Doody, L. Golbe, Erik D. Roberson, Mary Koestler, Clifford R. Jack, Viviana Van Deerlin, Christopher Randolph, Illana Gozes, Steve Whitaker, Joe Hirman, Michael Gold, Bruce H. Morimoto, J. C. Gómez, B. Tijero, K. Berganzo, J. García de Yebenes, J. L. Lopez Sendón, G. Garcia, E. Tolosa, M. T. Buongiorno, N. Bargalló, J. A. Burguera, I. Martinez, J. Ruiz-Martínez, I. Narrativel, F. Vivancos, I. Ybot, M. Aguilar, P. Quilez, M. Boada, A. Lafuente, I. Hernandez, J. J. López-Lozano, M. Mata, A. Kupsch, A. Lipp, G. Ebersbach, T. Schmidt, K. Hahn, G. Hoglinger, M. Hollerhage, H. Reichmann, M. Wolz, C. Schneider, L. Klingelhofer, D. Berg, W. Maetzler, K. K. Srulijes, A. Ludolph, J. Kassubek, M. Steiger, K. Tyler, D. J. Burn, L. Morris, A. Lees, H. Ling, R. Hauser, T. McClain, D. Truong, S. Jenkins, I. Litvan, D. Houghton, J. Ferrara, Y. Bordelon, A. Gratiano, L. Golbe, M. Mark, R. Uitti, J. Ven Gerpen, Kailash Bhatia, Yvette M. Bordelon, Carlo Colosimo, Richard Dodel, Keith A. Josephs, Huw Morris, Ulrich Mueller, Dominic Paviour, Gerard Schellenberg, John Steele, John C. van Swieten, Jennifer Whitwell

Research output: Contribution to journalArticlepeer-review

26 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)742-747
Number of pages6
JournalMovement Disorders
Volume31
Issue number5
DOIs
StatePublished - May 1 2016

Bibliographical note

Publisher Copyright:
© 2016 International Parkinson and Movement Disorder Society.

Keywords

  • Clinical trials
  • Placebo effect
  • Power calculation
  • Progressive supranuclear palsy
  • Rate of progression

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