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Table 3 Efficacy measures at baseline and week 8 in ITT population for Adolescent/adult study

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

Measure Placebo N = 63 (62 completers) Arbaclofen N = 62 (57 completers)
Baseline Week 8 Change Baseline Week 8 Change p
ABC-CFX SA 7.9 (3.48) 5.5 (3.37) −2.4 (0.32) 7.6 (3.17) 5.3 (3.57) −2.3 (0.33) 0.974
ABC-CFX I 15.8 (13.18) 10.8 (11.01) −5.3 (0.88) 16.9 (14.58) 12.3 (12.35) −4.2 (0.89) 0.421
ABC-CFX H 11.2 (7.32) 8.0 (6.75) −3.4 (0.57) 12.9 (8.44) 8.7 (6.82) −3.6 (0.58) 0.811
ABC-CFX SB 8.4 (5.77) 6.0 (5.30) −2.5 (0.40) 8.8 (5.37) 5.7 (4.84) −2.9 (0.40) 0.508
ABC-CFX L 12.3 (6.70) 7.3 (5.38) −5.2 (0.56) 12.8 (7.19) 7.9 (5.94) −4.7 (0.57) 0.536
ABC-CFX IS 6.4 (3.74) 4.6 (3.02) −1.8 (0.28) 6.5 (3.90) 4.8 (3.53) −1.5 (0.29) 0.452
CGI-I 3.1 (0.12) 3.2 (0.12) 0.587
CGI-S 4.7 (0.92) 4.4 (0.98) −0.3 (0.08) 4.6 (1.07) 4.1 (1.06) −0.5 (0.08) 0.063
Responder 25.00% 24.10% 0.913
PSI 126.5 (20.45) 129.1 (23.26) 4.1 (2.23) 115.4 (22.04) 120.2 (22.48) 3.3 (2.27) 0.816
VAS-Anx 59.9 (24.57) 39.1 (26.82) −21.2 (33.31) 62.0 (27.00) 46.4 (29.96) −14.7 (3.38) 0.176
VAS-Dis 32.9 (28.75) 27.4 (24.74) −7.0 (2.67) 37.6 (33.40) 33.3 (29.57) −3.9 (2.72) 0.413
Vineland-II Soc 55.2 (20.33) 57.4 (19.70) 2.5 (1.17) 53.4 (15.80) 53.5 (18.17) 0.1 (1.17) 0.151
Vineland-II Comm 51.4 (20.91) 53.4 (20.56) 1.9 (1.06) 48.5 (18.54) 48.9 (19.72) 0.2 (1.06) 0.274
Vineland-II Mal 19.6 (1.43) 19.1 (1.27) −0.6 (0.16) 20.0 (1.77) 19.2 (1.77) −0.7 (0.16) 0.638
CSHQ-T 42.4 (6.68) 41.1 (5.83) −1.3 (0.44) 42.7 (7.55) 41.1 (6.60) −1.4 (0.45) 0.857
CSHQ-DS 10.8 (2.26) 10.7 (2.49) −0.2 (0.25) 11.0 (2.83) 10.7 (2.49) −0.3 (0.26) 0.700
  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. ABC-C FX Aberrant Behavior Checklist 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, Vineland-II Soc Vineland Adaptive Behavior Scales, Second Edition Socialization domain standard score, Vineland-II Comm Vineland Adaptive Behavior Scales, Second Edition Communication domain standard score, Vineland-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