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

  • Author(s): Stamelou, M
  • Schöpe, J
  • Wagenpfeil, S
  • Del Ser, T
  • Bang, J
  • Lobach, IY
  • Luong, P
  • Respondek, G
  • Oertel, WH
  • Boxer, A
  • Höglinger, GU
  • Williams, D
  • Lafontaine, AL
  • Marras, C
  • Jog, M
  • Panisset, M
  • Lang, A
  • Parker, L
  • Stewart, AJ
  • Corvol, JC
  • Azulay, JP
  • 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
  • Gómez, JC
  • Tijero, B
  • Berganzo, K
  • García de Yebenes, J
  • Lopez Sendón, JL
  • Garcia, G
  • Tolosa, E
  • Buongiorno, MT
  • Bargalló, N
  • Burguera, JA
  • Martinez, I
  • Ruiz-Martínez, J
  • Narrativel, I
  • et al.

Published Web Location

https://doi.org/10.1002/mds.26580
Abstract

© 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.

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