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

Tau Restoration on PSP (TAUROS) Investigators, The MDS-Endorsed PSP Study Group

Research output: Contribution to journalArticle

9 Citations (Scopus)

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

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Progressive Supranuclear Palsy
Placebo Effect
Clinical Trials
Activities of Daily Living
England
Placebos
Sample Size
Randomized Controlled Trials

Keywords

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

ASJC Scopus subject areas

  • Clinical Neurology
  • Neurology

Cite this

Tau Restoration on PSP (TAUROS) Investigators, & The MDS-Endorsed PSP Study Group (2016). Power calculations and placebo effect for future clinical trials in progressive supranuclear palsy. Movement Disorders, 31(5), 742-747. https://doi.org/10.1002/mds.26580

Power calculations and placebo effect for future clinical trials in progressive supranuclear palsy. / Tau Restoration on PSP (TAUROS) Investigators; The MDS-Endorsed PSP Study Group.

In: Movement Disorders, Vol. 31, No. 5, 01.05.2016, p. 742-747.

Research output: Contribution to journalArticle

Tau Restoration on PSP (TAUROS) Investigators & The MDS-Endorsed PSP Study Group 2016, 'Power calculations and placebo effect for future clinical trials in progressive supranuclear palsy', Movement Disorders, vol. 31, no. 5, pp. 742-747. https://doi.org/10.1002/mds.26580
Tau Restoration on PSP (TAUROS) Investigators, The MDS-Endorsed PSP Study Group. Power calculations and placebo effect for future clinical trials in progressive supranuclear palsy. Movement Disorders. 2016 May 1;31(5):742-747. https://doi.org/10.1002/mds.26580
Tau Restoration on PSP (TAUROS) Investigators ; The MDS-Endorsed PSP Study Group. / Power calculations and placebo effect for future clinical trials in progressive supranuclear palsy. In: Movement Disorders. 2016 ; Vol. 31, No. 5. pp. 742-747.
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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.",
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AU - Wagenpfeil, Stefan

AU - Del Ser, Teodoro

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AU - Boxer, Adaml

AU - Höglinger, Günter U.

AU - Williams, David

AU - Lafontaine, Anne Louise

AU - Marras, Connie

AU - Jog, Mandar

AU - Panisset, Michael

AU - Lang, Anthony

AU - Parker, Lesley

AU - Stewart, Alistair J.

AU - Corvol, Jean Christophe

AU - Azulay, Jean Philippe

AU - Couratier, Philippe

AU - Mollenhauer, Brit

AU - Lorenzl, Stefan

AU - Ludolph, A.

AU - Benecke, Reiner

AU - Hoglinger, G.

AU - Lipp, A.

AU - Reichmann, H.

AU - Woitalla, Dirk

AU - Chan, Dennis

AU - Zermansky, Adam

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AU - Lees, A.

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AU - Honig, Lawrence

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AU - Bordelon, Y.

AU - Driver-Dunkley, Erika

AU - Lessig, Stephanie

AU - Lew, Mark

AU - Womack, Kyle

AU - Boeve, Brad

AU - Ferrara, J.

AU - Hillis, Argyle

AU - Kaufer, Daniel

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KW - Rate of progression

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