Table 2.
Primary and secondary outcome analysis
| 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 |
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
ADHD attention-deficit hyperactivity disorder, Lsmean least squares mean; SE standard error