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Mov Disord. 2016 May;31(5):742-7. doi: 10.1002/mds.26580. Epub 2016 Mar 7.

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

Author information

1
Department of Neurology, Philipps University, Marburg, Germany.
2
Second Department of Neurology, Attikon University Hospital, University of Athens, Athens, Greece.
3
Movement Disorders Department, Hygeia Hospital, Athens, Greece.
4
Institute for Medical Biometry, Epidemiology and Medical Informatics, Saarland University, Campus Homburg, Homburg, Germany.
5
Medical Department, Noscira SA, Madrid, Spain.
6
Alzheimer Project Research Unit, Fundación CIEN, Madrid, Spain.
7
Memory and Aging Center, Department of Neurology, University of California, San Francisco, California, USA.
8
Department of Neurology, Technische Universität München, Munich, Germany.
9
German Center for Neurodegenerative Diseases (DZNE), Munich, Germany.

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. © 2016 International Parkinson and Movement Disorder Society.

KEYWORDS:

clinical trials; placebo effect; power calculation; progressive supranuclear palsy; rate of progression

PMID:
26948290
PMCID:
PMC5289149
DOI:
10.1002/mds.26580
[Indexed for MEDLINE]
Free PMC Article

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