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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
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Journal of Neurodevelopmental Disorders

ISSN: 1866-1955

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