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. 2016 Aug 26;20(1):11–21. doi: 10.1093/ijnp/pyw076

Table 2.

Secondary Efficacy Endpoints

Single-Blind Stabilization Phase (Stabilization Phase Efficacy Sample; n=449) Double-Blind Maintenance Phase (Maintenance Phase efficacy sample; n=200)
Baseline Mean (SD) Mean (SD) Change at Last (Post-Baseline) Visit Baseline Mean (SD) MMRM analysis LOCF, ANCOVA
LS Mean Change (SE) at Week 52 Treatment Difference (95% CI) P Value LS Mean Change (SE) at Week 52 Treatment Difference (95% CI) P Value
Brex Brex Brex Placebo Brex Placebo Brex Placebo
PANSS total score 84.26 (12.33) -15.13 (15.99) 56.66 (8.51) 58.07 (8.13) 0.61 (3.34) 6.92 (4.53) -6.31 (-18.1, 5.46) .2800 3.25 (1.94) 11.20 (1.77) -7.95 (-12.5, -3.41) .0007
[n=449] [n=447] [n=96] [n=104] [n=15] [n=9] [n=96] [n=104]
PANSS positive subscale score 21.35 (4.75) -4.99 (5.45) 12.48 (3.66) 12.63 (3.13) -1.21 (0.73) 1.50 (0.99) -2.71 (-5.20, -0.22) .0339 0.99 (0.64) 4.17 (0.59) -3.18 (-4.70, -1.66) <.0001
[n=449] [n=447] [n=96] [n=104] [n=15] [n=9] [n=96] [n=104]
PANSS negative subscale score 22.14 (4.64) -3.16 (4.61) 16.30 (3.46) 17.00 (3.64) 1.30 (1.37) 0.87 (1.71) 0.43 (-4.14, 5.00) .8470 0.39 (0.54) 1.63 (0.49) -1.24 (-2.50, 0.01) .0516
[n=449] [n=447] [n=96] [n=104] [n=15] [n=9] [n=96] [n=104]
CGI-S score 4.31 (0.78) -0.73 (0.96) 3.00 (0.60) 3.08 (0.60) -0.23 (0.17) 0.28 (0.22) -0.51 (-1.09, 0.06) .0780 0.02 (0.11) 0.55 (0.11) -0.53 (-0.79, -0.26) .0002
[n=449] [n=449] [n=96] [n=104] [n=15] [n=9] [n=96] [n=104]
CGI-I score NA 2.84 (1.24) a NA NA - - - - 3.77 (1.26)b 4.40 (1.32) b -0.61 (-0.96, -0.25) .0009
[n=454] [n=96] [n=104]
PSP score - - 49.82 (12.20) 48.22 (11.64) 18.63 (2.76) 12.55 (3.48) 6.08 (-2.71, 14.87) .1677 15.06 (1.43) 10.31 (1.34) 4.75 (1.31, 8.18) .0071
[n=94] [n=100] [n=15] [n=9] [n=94] [n=100]
GAF score 45.82 (10.34) 11.42 (12.30) 64.22 (9.00) 63.00 (8.31) 5.72 (1.87) -0.16 (2.37) 5.88 (-0.06, 11.82) .0522 0.55 (1.38) -6.01 (1.28) 6.55 (3.28, 9.83) .0001
[n=449] [n=426] [n=95] [n=102] [n=15] [n=9] [n=95] [n=102]
PANSS excited component score 11.03 (3.44) -1.89 (3.97) 6.98 (1.92) 7.37 (2.50) -0.04 (0.46) 1.00 (0.59) -1.03 (-2.58, 0.51) .1803 0.82 (0.41) 2.35 (0.38) -1.54 (-2.52, -0.56) .0023
[n=449] [n=447] [n=96] [n=104] [n=15] [n=9] [n=96] [n=104]
PANSS Marder factor scores
Positive symptoms 25.46 (5.13) -5.48 (5.66) 15.89 (3.99) 15.80 (3.21) -1.62 (0.83) 1.78 (1.05) -3.40 (-6.05, -0.75) .0136 0.58 (0.66) 4.02 (0.60) -3.44 (-4.99, -1.89) <.0001
Negative symptoms 21.05 (4.92) -3.23 (4.81) 15.07 (3.53) 16.06 (3.68) 1.37 (1.39) 1.06 (1.77) 0.31 (-4.40, 5.02) .8927 0.34 (0.56) 1.57 (0.50) -1.23 (-2.52, 0.07) .0630
Disorganized thought 19.98 (3.86) -3.42 (3.95) 14.54 (2.89) 14.31 (3.06) -0.37 (0.99) -0.30 (1.24) -0.07 (-3.32, 3.17) .9632 0.29 (0.48) 1.97 (0.45) -1.69 (-2.81, -0.56) .0035
Uncontrolled hostility/ excitement 8.58 (3.14) -1.42 (3.35) 5.56 (1.76) 5.72 (2.17) -0.20 (0.40) 0.94 (0.50) -1.14 (-2.46, 0.18) .0875 0.49 (0.37) 1.75 (0.34) -1.26 (-2.12, -0.39) .0046
Anxiety/depression 9.18 (3.21) -1.57 (3.42) 5.59 (1.76) 6.18 (2.47) 0.04 (0.43) 0.28 (0.56) -0.23 (-1.68, 1.21) .7437 1.17 (0.31) 1.88 (0.29) -0.72 (-1.47, 0.03) .0608
[n=449] [n=447] [n=96] [n=104] [n=15] [n=9] [n=96] [n=104]
Cogstate cognitive test battery Last (post-baseline) visit, ANCOVA
Composite change score c 0.09 (0.88) -0.12 (0.70) 0.10 (0.89) 0.11 (0.88) - - - - 0.06 (0.07) -0.13 (0.07) 0.19 (0.01, 0.37) .0419
[n=441] [n=161] [n=82] [n=82] [n=82] [n=82]
Detection task (log10 msec) d 2.66 (0.17) 0.02 (0.13) 2.68 (0.17) 2.67 (0.17) - - - - 0.01 (0.01) 0.01 (0.01) -0.01 (-0.04, 0.03) .7724
[n=444] [n=164] [n=82] [n=82] [n=82] [n=82]
Identification task (log10 msec)d 2.79 (0.12) 0.01 (0.10) 2.80 (0.12) 2.80 (0.12) - - - - -0.01 (0.01) 0.02 (0.01) -0.03 (-0.06, 0.00) .0312
[n=444] [n=164] [n=84] [n=83] [n=84] [n=83]
One-card learning task c 0.92 (0.17) -0.01 (0.16) 0.95 (0.18) 0.94 (0.16) - - - - 0.02 (0.02) -0.04 (0.02) 0.06 (0.01, 0.11) .0321
[n=447] [n=167] [n=84] [n=85] [n=84] [n=85]
Groton maze learning task d 72.5 (46.9) 0.4 (68.3) 68.24 (36.15) 63.21 (27.34) - - - - 0.22 (3.26) 2.43 (3.34) -2.21 (-11.4, 7.02) .6371
[n=432] [n=163] [n=84] [n=80] [n=84] [n=80]

Abbreviations: Brex, brexpiprazole 1–4mg; CGI-I, Clinical Global Impressions – Improvement; CGI-S, Clinical Global Impressions – Severity of illness; GAF, Global Assessment of Functioning; LOCF, last observation carried forward; LS, least squares; MMRM, mixed-effect model repeated measures; NA, not applicable; PANSS, Positive and Negative Syndrome Scale; PSP, Personal and Social Performance; –, not assessed/analysis not performed.

aMean (SD) score at patients’ last (post-baseline) visit.

bMean (SD) score at week 52.

cAn increase in score indicates improvement.

dA decrease in score indicates improvement.

In the maintenance phase, MMRM analyses using observed data (treatment, trial center, visit, treatment–visit interaction, and baseline value as fixed effects; baseline–visit interaction as covariate) were performed, along with LOCF sensitivity analyses using an ANCOVA model (treatment and trial center as factors; baseline value as covariate). In the maintenance phase, CGI-I score was analyzed using the Cochran–Mantel–Haenszel method with LOCF data. For Cogstate analyses in the maintenance phase, an ANCOVA model (treatment as factor; baseline value as covariate) was used.