Table 2 Change from Baseline at Week 12 for all Efficacy Endpoints

From: Effect of the mGluR5-NAM Basimglurant on Behavior in Adolescents and Adults with Fragile X Syndrome in a Randomized, Double-Blind, Placebo-Controlled Trial: FragXis Phase 2 Results

Assessment (analysis method)

Placebo

Basimglurant 0.5 mg

Basimglurant 1.5 mg

 

n

Mean (SEM)

n

Mean (SEM)

n

Mean (SEM)

ADAMS Total Score (MMRM)

61

−10.63 (1.49)a

51

−6.20 (1.69)

51

−8.63 (1.55)

Demographic subgroup analysis

 Adolescents (<18 years)

21

−9.4 (2.3)

17

−4.4 (2.7)

18

−8.4 (2.5)

 Adults (⩾18 years)

40

−11.8 (1.8)

34

−7.7 (1.9)

33

−9.2 (1.9)

 Males

50

−9.1 (1.5)

43

−6.3 (1.7)

42

−9.0 (1.6)

 Females

11

−18.9 (2.9)

8

−6.8 (3.5)

9

−8.4 (3.1)

 Antipsychotic use

13

−4.0 (2.8)

12

0.0 (2.8)

10

−4.4 (2.9)

 No antipsychotic use

48

−12.0 (1.5)

39

−7.6 (1.8)

41

−9.3 (1.6)

Biomarker subgroup analyses

 Methylation positive (⩾73.95%)

22

−9.3 (2.3)

23

−6.7 (2.3)

23

−9.2 (2.3)

 Methylation negative (<73.95%)

39

−11.5 (1.9)a

28

−5.8 (2.2)

28

−8.7 (1.9)

 mRNA Δ count positive (⩾1.975)

25

−7.2 (3.2)

22

−3.4 (3.4)

23

−5.6 (3.0)

 mRNA Δ count negative (<1.975)

35

−11.6 (1.9)

28

−6.3 (2.1)

27

−9.5 (2.0)

 FMRP positive (⩽0.146 pM)

18

−7.1 (2.3)

19

−6.1 (2.4)

22

−8.1 (2.3)

 FMRP negative (>0.146 pM)

34

−13.4 (2.2)

21

−5.6 (2.8)

25

−9.4 (2.3)

ABC Total (MMRM)

62

−16.26 (2.81)

51

−10.46 (3.11)

51

−11.53 (2.91)

CGI-S (Wilcoxon-LOCF)

62

−0.26 (0.07)

58

−0.26 (0.08)

56

−0.27 (0.10)

CGI-I (Wilcoxon-LOCF)

62

3.06 (0.11)a

58

3.47 (0.12)

57

3.39 (0.16)

Clinical Response Rate (Fisher-LOCF)b

63

23.81%

58

13.79%

62

17.74%

SRS T-Score (MMRM)

62

−8.25 (1.44)c

53

−3.65 (1.58)

52

−4.65 (1.48)

RBANS Immediate Memory (ANCOVA-LOCF)

59

0.69 (1.52)

50

1.37 (1.67)

46

0.87 (1.57)

VABS-II Composite Standard Score (MMRM)

47

3.93 (2.57)

40

1.70 (2.80)

41

2.71 (2.50)

VAS MTS (Wilcoxon-LOCF)

62

−20.35 (4.03)

56

−12.45 (3.34)

54

−16.11 (4.20)

  1. A decrease in ADAMS, ABC, CGI-S, CGI-I, SRS, and VAS scores indicates improvement. An increase in the RBANS and VABS-II score indicates improvement. Values are least square mean (SEM).
  2. Analyses conducted were based upon the assessment and include Mixed Effect Model Repeated Measures (MMRM), Wilcoxon-Last Observation Carried Forward (LOCF), Fisher-LOCF, and analysis of covariance (ANCOVA)-LOCF, all with standard error of the mean (SEM).
  3. ap ⩽0.05 compared to basimglurant 0.5 mg.
  4. bClinical response defined as a composite of improvement in ABC Total Score of 25% or more and of a CGI-I score of either 1 or 2.
  5. cp ⩽0.05 compared to basimglurant 0.5 and 1.5 mg.
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