. 2023 Nov 6;80(12):1307–1316. doi: 10.1001/jamaneurol.2023.3810

Table 2. Summary of Efficacy End Points (Efficacy Sample).

End point	Treatment group	No. of patients	Baseline, mean (SD)	At week 12, change from baseline, LS mean (SE), or No. (%) response rate	Treatment difference at week 12 vs placebo
End point	Treatment group	No. of patients	Baseline, mean (SD)		LS mean difference or ratio of response rate (95% CI)	P value	Cohen d effect size
Primary end point
CMAI total score^a	Brexpiprazole 2 or 3 mg	225	80.6 (16.6)	−22.6 (1.1)	Difference, −5.32 (−8.77 to −1.87)	.003	0.35
CMAI total score^a	Placebo	116	79.2 (17.5)	−17.3 (1.4)	Difference, −5.32 (−8.77 to −1.87)	.003	0.35
Key secondary end point
CGI-S score as related to agitation^a	Brexpiprazole 2 or 3 mg	225	4.7 (0.7)	−1.2 (0.1)	Difference, −0.27 (−0.47 to −0.07)	.008	0.31
CGI-S score as related to agitation^a	Placebo	116	4.7 (0.7)	−0.9 (0.1)	Difference, −0.27 (−0.47 to −0.07)	.008	0.31
Secondary end points: change and difference ^b
CMAI factor 1: aggressive behavior score^a	Brexpiprazole 2 or 3 mg	225	26.3 (7.3)	−9.1 (0.4)	Difference, −1.95 (−3.28 to −0.63)	.004	0.33
CMAI factor 1: aggressive behavior score^a	Placebo	116	26.5 (8.7)	−7.1 (0.6)	Difference, −1.95 (−3.28 to −0.63)	.004	0.33
CMAI factor 2: physically non-aggressive behavior score^a	Brexpiprazole 2 or 3 mg	225	23.8 (7.3)	−6.5 (0.4)	Difference, −1.41 (−2.68 to −0.14)	.03	0.25
CMAI factor 2: physically non-aggressive behavior score^a	Placebo	116	23.2 (7.4)	−5.0 (0.5)	Difference, −1.41 (−2.68 to −0.14)	.03	0.25
CMAI factor 3: verbally agitated behavior score^a	Brexpiprazole 2 or 3 mg	225	16.9 (4.7)	−4.4 (0.3)	Difference, −1.24 (−2.21 to −0.28)	.01	0.29
CMAI factor 3: verbally agitated behavior score^a	Placebo	116	16.3 (5.6)	−3.1 (0.4)	Difference, −1.24 (−2.21 to −0.28)	.01	0.29
CGI-I score^c	Brexpiprazole 2 or 3 mg	225	NA	2.7 (1.1)^d	Difference, −0.33 (−0.57 to −0.09)^e	.007	NA
CGI-I score^c	Placebo	116	NA	3.0 (1.1)^d	Difference, −0.33 (−0.57 to −0.09)^e	.007	NA
Secondary end points: response rate and ratio ^b
CMAI response rate^c
≥20% Improvement	Brexpiprazole 2 or 3 mg	225	NA	154 (68.4)	Ratio, 1.41 (1.15 to 1.72)^f	<.001	NA
≥20% Improvement	Placebo	116	NA	55 (47.4)	Ratio, 1.41 (1.15 to 1.72)^f	<.001	NA
≥30% Improvement	Brexpiprazole 2 or 3 mg	225	NA	96 (42.7)	Ratio, 1.62 (1.18 to 2.23)^f	.002	NA
≥30% Improvement	Placebo	116	NA	30 (25.9)	Ratio, 1.62 (1.18 to 2.23)^f	.002	NA
≥40% Improvement	Brexpiprazole 2 or 3 mg	225	NA	52 (23.1)	Ratio, 1.62 (1.00 to 2.61)^f	.03	NA
≥40% Improvement	Placebo	116	NA	17 (14.7)	Ratio, 1.62 (1.00 to 2.61)^f	.03	NA
Improvement in agitation status	Brexpiprazole 2 or 3 mg	225	NA	118 (52.4)	Ratio, 1.47 (1.14 to 1.89)^f	.002	NA
Improvement in agitation status	Placebo	116	NA	43 (37.1)	Ratio, 1.47 (1.14 to 1.89)^f	.002	NA
CGI-I response rate^c
Score of very much improved or much improved	Brexpiprazole 2 or 3 mg	225	NA	118 (52.4)	Ratio, 1.32 (1.03 to 1.69)^f	.02	NA
Score of very much improved or much improved	Placebo	116	NA	47 (40.5)	Ratio, 1.32 (1.03 to 1.69)^f	.02	NA
Exploratory end point ^b
NPI-NH total score^a	Brexpiprazole 2 or 3 mg	215	37.7 (17.8)	−17.3 (0.9)	Difference, −4.60 (−7.33 to −1.88)	.001	0.39
NPI-NH total score^a	Placebo	111	36.6 (17.2)	−12.7 (1.2)	Difference, −4.60 (−7.33 to −1.88)	.001	0.39

Abbreviations: CGI-I, Clinical Global Impression–Improvement; CGI-S, Clinical Global Impression–Severity of illness; CMAI, Cohen-Mansfield Agitation Inventory; CMH, Cochran-Mantel-Haenszel; LS, least-squares; NA, not applicable; NPI-NH, Neuropsychiatric Inventory–Nursing Home version.

^{^a}

Mixed model for repeated measures.

^{^b}

P values for secondary and exploratory end points are nominal with no adjustment for multiplicity.

^{^c}

Last observation carried forward.

^{^d}

Mean (SD) score at week 12.

^{^e}

Adjusted mean difference (95% CI) based on the CMH row mean scores differ test.

^{^f}

Brexpiprazole/placebo; CMH general association test.