Title |
Power calculations and placebo effect for future clinical trials in progressive supranuclear palsy
|
---|---|
Published in |
Movement Disorders, March 2016
|
DOI | 10.1002/mds.26580 |
Pubmed ID | |
Authors |
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, Tauros Investigators for the AL‐108‐231 Investigators |
Abstract |
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. We provide sample size calculations based on data collected in 187 PSP patients assigned to placebo in these trials. A placebo effect was calculated. 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. 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. |
X Demographics
Geographical breakdown
Country | Count | As % |
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United States | 3 | 100% |
Demographic breakdown
Type | Count | As % |
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Practitioners (doctors, other healthcare professionals) | 1 | 33% |
Members of the public | 1 | 33% |
Science communicators (journalists, bloggers, editors) | 1 | 33% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
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Philippines | 1 | <1% |
Germany | 1 | <1% |
Unknown | 106 | 98% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Other | 20 | 19% |
Researcher | 15 | 14% |
Professor | 12 | 11% |
Student > Ph. D. Student | 8 | 7% |
Student > Bachelor | 7 | 6% |
Other | 21 | 19% |
Unknown | 25 | 23% |
Readers by discipline | Count | As % |
---|---|---|
Medicine and Dentistry | 34 | 31% |
Neuroscience | 24 | 22% |
Psychology | 4 | 4% |
Nursing and Health Professions | 3 | 3% |
Pharmacology, Toxicology and Pharmaceutical Science | 2 | 2% |
Other | 10 | 9% |
Unknown | 31 | 29% |