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Table 4 Efficacy measures at baseline and week 8 in ITT population for child study

From: Arbaclofen in fragile X syndrome: results of phase 3 trials

Measure Placebo N = 45 (44 completers) Arbaclofen 5 mg BID N = 41 (38 completers) Arbaclofen 10 mg BID N = 41 (39 completers) Arbaclofen 10 mg TID N = 42 (38 completers)
Baseline Week 8 Change Baseline Week 8 Change p Baseline Week 8 Change p Baseline Week 8 Change p
ABC-CFX SA 6.9 (3.49) 4.2 (3.24) −2.8 (0.36) 6.9 (2.84) 3.8 (2.46) −3.1 (0.38) 0.488 6.9 (3.03) 4.1 (2.85) −2.8 (0.38) 0.968 6.4 (2.92) 2.9 (2.72) −3.7 (0.38) 0.085
ABC-CFX I 27.8 (12.91) 22.4 (14.53) −5.5 (1.31) 29.7 (11.16) 21.7 (9.71) −8.6 (1.41) 0.112 28.0 (10.81) 19.5 (11.81) −8.8 (1.41) 0.112 30.4 (9.53) 21.6 (10.95) −9.7 (1.41) 0.031
ABC-CFX H 19.6 (7.82) 15.7 (8.28) −4.0 (0.75) 21.6 (6.93) 17.2 (7.31) −4.5 (0.81) 0.665 19.4 (6.58) 15.1 (7.57) −4.6 (0.81) 0.577 21.9 (5.29) 15.9 (7.22) −6.0 (0.81) 0.081
ABC-CFX SB 9.3 (5.81) 6.3 (5.52) −3.1 (0.50) 11.0 (4.81) 7.3 (4.26) −3.5 (0.54) 0.613 9.6 (4.77) 6.7 (4.64) −3.0 (0.54) 0.884 11.2 (5.01) 7.9 (5.12) −3.2 (0.54) 0.908
ABC-CFX L 11.6 (6.48) 7.3 (4.99) −4.9 (0.64) 12.8 (6.05) 7.4 (5.03) −5.4 (0.69) 0.577 13.3 (5.40) 8.0 (5.09) −5.1 (0.69) 0.837 13.2 (5.08) 7.2 (5.94) −5.8 (0.69) 0.324
ABC-CFX IS 6.2 (3.86) 4.9 (3.76) −1.5 (0.36) 7.2 (3.51) 5.3 (3.48) −2.2 (0.38) 0.210 7.5 (3.52) 5.7 (3.93) −1.6 (0.38) 0.868 7.9 (3.54) 6.1 (3.43) −1.6 (0.38) 0.791
CGI-I 3.3 (1.00) 3.3 (1.16) 0.993 3.1 (0.96) 0.320 2.9 (0.87) 0.119
CGI-S 4.8 (0.79) 4.3 (1.01) −0.4 (0.10) 5.1 (1.00) 4.7 (0.99) −0.5 (0.11) 0.606 5.0 (0.89) 4.5 (0.91) −0.4 (0.11) 0.700 4.9 (0.81) 4.4 (0.86) −0.5 (0.11) 0.909
Responder 22.00% 26.30% 0.650 29.70% 0.432 35.10% 0.196
PSI 117.0 (21.97) 119.6 (19.65) 3.4 (1.96) 110.8 (17.46) 116.2 (15.34) 4.2 (2.18) 0.781 119.5 (20.07) 122.9 (18.83) 5.2 (2.11) 0.537 116.3 (18.97) 125.4 (20.93) 9.7 (2.16) 0.032
VAS-Anx 73.6 (18.99) 54.7 (25.54) −19.1 (3.45) 75.0 (17.64 57.2 (24.05) −18.8 (3.74) 0.957 77.7 (15.1) 55.8 (26.01) −20.2 (3.71) 0.828 70.2 (16.37) 51.4 (27.43) −18.8 (3.75) 0.950
VAS-Dis 58.0 (27.47) 47.2 (28.20) −11.9 (3.31) 61.9 (25.83) 51.4 (25.59) −10.6 (3.59) 0.787 61.5 (22.28) 46.6 (25.59) −14.4 (3.54) 0.601 65.6 (23.69) 51.4 (27.10) −10.7 (3.58 0.809
V-II Soc 64.0 (13.08) 66.0 (9.78) 3.1 (1.10) 64.8 (14.29) 68.9 (14.47) 3.6 (1.10) 0.191 67.5 (13.43) 69.3 (13.12) 1.9 (1.08) 0.866 64.5 (14.58) 66.7 (13.41) 3.1 (1.10) 0.314
V-II Comm 65.9 (12.92) 67.9 (10.79) 1.2 (0.72) 65.0 (12.51) 67.2 (10.78) 1.4 (0.80) 0.877 69.5 (14.81) 69.8 (14.89) 0.5 (0.78) 0.477 65.7 (11.88) 66.9 (11.88) 1.4 (0.79) 0.915
V-II Mal 20.2 (1.87) 19.5 (1.78) −0.7 (0.18) 20.6 (1.31) 19.8 (1.42) −0.7 (0.20) 0.877 19.8 (1.44) 19.3 (1.56) 0.6 (0.20) 0.667 20.5 (1.30) 19.4 (1.90) −1.0 (0.20) 0.246
CSHQ-T 45.9 (7.98) 45.0 (7.41) −1.5 (0.75) 47.3 (7.12) 46.3 (7.17) −1.2 (0.76) 0.851 45.0 (6.97) 45.7 (7.60) −0.1 (0.75) 0.402 47.2 (7.77) 45.9 (7.25) −1.5 (0.75) 0.640
CSHQ-DS 11.1 (2.53) 11.1 (2.70) −0.1 (0.28) 11.2 (2.83) 10.8 (2.43) −0.5 (0.31) 0.310 11.2 (2.89) 10.9 (2.63) −0.4 (0.30) 0.445 11.3 (2.70) 11.1 (2.12) −0.3 (0.30) 0.619
  1. Completers are those who finished the 8 week treatment period and assessments. All baseline, week 8 and change values given as mean (SE) for the group, except responder values which are given as percent responders out of total group, p values are for adjusted mean changes relative to the placebo group and adjusted mean changes are shown in the table
  2. BID twice daily, TID three times daily, ABC-C FX Aberrant Behavior Checklist-Community Edition refactored for FXS, SA Social Avoidance subscale, I Irritability subscale, H Hyperactivity subscale, SB Stereotypic Behavior subscale, L Socially Unresponsive/Lethargic subscale, IS Inappropriate Speech subscale, CGI-I Clinician Global Impression of Improvement, CGI-S Clinician Global Impression of Severity, Responder percent of participants with at least a 25% improvement on the ABC-CFX Social Avoidance primary outcome and a CGI-I of 1 (very much improved) or 2 (much improved) at 8 weeks, PSI Parenting Stress Index, VAS-Anx visual analog scale for Anxiety, VAS – Dis visual analog scale for Disruptive Behaviors, V-II Soc Vineland Adaptive Behavior Scales, Second Edition – Socialization domain standard score, V-II Comm Vineland Adaptive Behavior Scales, Second Edition – Communication domain standard score, V-II Mal Vineland Adaptive Behavior Scales, Second Edition – Maladaptive Behavior Index standard score, CSHQ-T Children’s Sleep Habits Questionnaire – Total score, CSHQ-DS Children’s Sleep Habits Questionnaire - Daytime Sleepiness subscale