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Table 2 Primary and secondary outcome analysis

From: A randomized double-blind, placebo-controlled trial of ganaxolone in children and adolescents with fragile X syndrome

  Ganaxolone Placebo  
Baseline End of treatment Baseline End of treatment
N Mean SE Lsmean SE N Mean SE Lsmean SE p value
Primary outcome
 Clinical Global Impression-Improvement 52 3.4 0.13 54 3.5 0.13 0.45
Key secondary outcome
 Pediatric Anxiety Rating Scale (total score) 55 10.5 0.76 8.3 0.54 55 10.9 0.73 9.2 0.54 0.22
Other secondary outcome
 Visual Analogue Scale
  Severity of Anxiety (cm) 55 4.3 0.37 5.6 0.27 54 4.2 0.34 5.0 0.26 0.13
  Severity of attention (cm) 55 3.4 0.30 4.4 0.24 54 3.3 0.31 3.9 0.24 0.14
  Severity of one selected additional target behavior (sociability, language, aggression, hyperactivity/impulsivity) (cm) 55 3.2 0.30 4.4 0.28 54 3.3 0.33 3.9 0.27 0.25
Anxiety, Depression and Mood Scale
 Manic/hyperactive behavior total 54 8.1 0.51 7.4 0.42 53 8.7 0.50 7.8 0.40 0.49
 Depressed mood total 55 3.0 0.46 3.5 0.32 54 3.1 0.42 2.6 0.32 0.01
 Social avoidance total 55 6.9 0.60 5.8 0.42 54 7.5 0.62 6.3 0.41 0.39
 General anxiety total 55 7.9 0.62 6.2 0.45 54 8.3 0.60 7.0 0.44 0.21
 Obsessive/compulsive behavior total 55 3.0 0.32 2.7 0.21 54 3.4 0.31 2.8 0.20 0.81
Aberrant Behavior Checklist-Community Edition FXS algorithm (ABC-CFX)
 Total - subscale I (irritability) 55 18.9 1.53 15.7 0.87 54 19.6 1.51 16.1 0.86 0.62
 Total - subscale II (lethargy) 55 6.6 0.68 5.6 0.53 52 6.9 0.68 5.7 0.54 0.74
 Total - subscale III (stereotypy) 55 7.4 0.68 5.7 0.40 54 7.1 0.68 6.4 0.39 0.17
 Total - subscale IV (hyperactivity) 54 13.9 1.00 11.3 0.65 54 13.9 1.14 12.3 0.62 0.28
 Total - subscale V (inappropriate speech) 55 6.0 0.47 5.1 0.32 54 6.3 0.43 5.3 0.31 0.47
 Total - subscale VI (social avoidance) 55 3.5 0.43 2.4 0.22 54 3.1 0.40 2.8 0.21 0.25
Swanson, Nolan, and Pelham-IV Questionnaire
 ADHD inattention total 53 15.7 0.76 15.5 0.66 53 16.7 0.79 14.6 0.62 0.28
 ADHD inattention average 53 1.7 0.08 1.7 0.07 53 1.9 0.09 1.6 0.07 0.28
 ADHD hyperactive impulsive total 54 13.0 0.93 13.9 0.66 53 14.0 0.92 12.6 0.64 0.12
 ADHD hyperactive impulsive average 54 1.4 0.10 1.5 0.07 53 1.6 0.10 1.4 0.07 0.14
 ADHD combined total 53 28.7 1.52 29.3 1.19 53 30.7 1.54 27.1 1.14 0.15
 ADHD combined average 53 1.6 0.08 1.6 0.07 53 1.7 0.09 1.5 0.06 0.21
  1. Analysis of study measures in overall participant population (N = 55 patients who completed at least one period). There were no statistically significant differences in primary or key secondary measures. However, there was statistically significant increase (worsening) on the ADAMS depressed mood subscale
  2. ADHD attention-deficit hyperactivity disorder, Lsmean least squares mean; SE standard error