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

Επιστημονική δημοσίευση - Άρθρο Περιοδικού uoadl:2998557 40 Αναγνώσεις

Μονάδα:
Ερευνητικό υλικό ΕΚΠΑ
Τίτλος:
Power calculations and placebo effect for future clinical trials in progressive supranuclear palsy
Γλώσσες Τεκμηρίου:
Αγγλικά
Περίληψη:
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.
Έτος δημοσίευσης:
2016
Συγγραφείς:
Stamelou, M.
Schöpe, J.
Wagenpfeil, S.
Del Ser, T.
Bang, J.
Lobach, I.Y.
Luong, P.
Respondek, G.
Oertel, W.H.
Boxer, A.
Höglinger, G.U.
Williams, D.
Lafontaine, A.L.
Marras, C.
Jog, M.
Panisset, M.
Lang, A.
Parker, L.
Stewart, A.J.
Corvol, J.-C.
Azulay, J.-P.
Couratier, P.
Mollenhauer, B.
Lorenzl, S.
Ludolph, A.
Benecke, R.
Hoglinger, G.
Lipp, A.
Reichmann, H.
Woitalla, D.
Chan, D.
Zermansky, A.
Burn, D.J.
Lees, A.
Boxer, A.
Miller, B.L.
Lobach, I.V.
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, D.S.
Schneider, L.S.
Doody, R.S.
Golbe, L.
Koestler, M.
Jack, C.R.
Van Deerlin, V.
Randolph, C.
Gozes, I.
Whitaker, S.
Hirman, J.
Gold, M.
Morimoto, B.H.
Gómez, J.C.
Tijero, B.
Berganzo, K.
García de Yebenes, J.
Lopez Sendón, J.L.
Garcia, G.
Tolosa, E.
Buongiorno, M.T.
Bargalló, N.
Burguera, J.A.
Martinez, I.
Ruiz-Martínez, J.
Narrativel, I.
Vivancos, F.
Ybot, I.
Aguilar, M.
Quilez, P.
Boada, M.
Lafuente, A.
Hernandez, I.
López-Lozano, J.J.
Mata, M.
Kupsch, A.
Lipp, A.
Ebersbach, G.
Schmidt, T.
Hahn, K.
Hoglinger, G.
Hollerhage, M.
Reichmann, H.
Wolz, M.
Schneider, C.
Klingelhofer, L.
Berg, D.
Maetzler, W.
Srulijes, K.K.
Ludolph, A.
Kassubek, J.
Steiger, M.
Tyler, K.
Morris, L.
Lees, A.
Ling, H.
Hauser, R.
McClain, T.
Truong, D.
Jenkins, S.
Litvan, I.
Houghton, D.
Ferrara, J.
Bordelon, Y.
Gratiano, A.
Golbe, L.
Mark, M.
Uitti, R.
Ven Gerpen, J.
Bhatia, K.
Bordelon, Y.M.
Colosimo, C.
Dodel, R.
Josephs, K.A.
Morris, H.
Mueller, U.
Paviour, D.
Schellenberg, G.
Steele, J.
van Swieten, J.C.
Whitwell, J.
Tau Restoration on PSP (TAUROS) Investigators
The MDS-Endorsed PSP Study Group
Περιοδικό:
Movement Disorders
Εκδότης:
John Wiley and Sons Inc
Τόμος:
31
Αριθμός / τεύχος:
5
Σελίδες:
742-747
Λέξεις-κλειδιά:
davunetide; tideglusib; 4 benzyl 2 methyl 1,2,4 thiazolidine 3,5 dione; davunetide; oligopeptide; thiadiazole derivative, activity of daily living assessment; aged; Article; data analysis; disease course; disease duration; female; human; major clinical study; male; placebo effect; power analysis; priority journal; progressive supranuclear palsy; randomized controlled trial (topic); rating scale; sample size; Schwab and England Activities of Daily Living; daily life activity; methodology; outcome assessment; progressive supranuclear palsy; severity of illness index; statistics and numerical data, Activities of Daily Living; Humans; Oligopeptides; Outcome Assessment (Health Care); Placebo Effect; Randomized Controlled Trials as Topic; Research Design; Sample Size; Severity of Illness Index; Supranuclear Palsy, Progressive; Thiadiazoles
Επίσημο URL (Εκδότης):
DOI:
10.1002/mds.26580
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