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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
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Journal of Neurodevelopmental Disorders

ISSN: 1866-1955

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