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Table 2 Characteristics of early phase studies and associations with phase III study results

From: Analysis of early phase and subsequent phase III stroke studies of neuroprotectants: outcomes and predictors for success

Study characteristics All early phase studies (n = 59) True neutral (n = 37) False positive (n = 22) aOR [95%-CI] P
No. of subjects, median (IQR) 92.0 (46.0-176.0) 100.0 (46.0-157.0) 91.5 (49.3-197.5) 1.00 [0.99-1.00] 0.713
Industry sponsored, n (%) 36 (61.0) 25 (67.6) 11 (50.0) 1.73 [0.40-7.54] 0.465
Multicentered, n (%) 41 (69.5) 28 (75.7) 13 (59.1) 1.48 [0.28-7.87] 0.643
Randomized, n (%) 57 (96.6) 35 (94.6) 22 (100.0) 0.00 [0.00-∞] 1.000
Blinded outcome assessment, n (%) 52 (88.1) 34 (91.9) 18 (81.8) 3.95 [0.26-60.30] 0.323
Dose–response investigated, n (%) 16 (27.1) 12 (32.4) 4 (18.2) 1.25 [0.26-6.07] 0.786
Use of imaging endpoint, n (%) 15 (25.4) 8 (21.6) 7 (31.8) 0.46 [0.12-1.81] 0.263
Duration of follow-up for endpoints*, median (IQR), days 87.0 (30.0-90.0) 90.0* (30.0-90.0) 41.0 (28.0-90.0) 1.00 [0.99-1.01] 0.722
Same characteristics in phase I/II studies as in subsequent phase III studies      
 Same therapeutic time window, n (%) 19 (32.2) 10 (27.0) 9 (40.9) 0.69 [0.18-2.68] 0.588
 Same dose, n (%) 29 (49.2) 18 (48.6) 11 (50.0) 0.94 [0.28-3.14] 0.916
 Same route of administration, n (%) 50 (84.7) 34 (91.9) 16 (72.7) 3.33 [0.59-18.86] 0.174
  1. IQR, interquartile range, aOR (adjusted odds ratio) for comparison of study characteristics between “false positive” and “true neutral” studies in a multivariable logistic regression analysis, CI, confidence interval, *one study did not report the exact duration of follow-up as endpoints were determined at discharge (mean length of hospital stay in this study was 9.5 days in the verum group and 11.2 days in the placebo group).