Skip to main content

Advertisement

Table 3 Change in primary and secondary efficacy measures from screening to week 12

From: A phase 1/2, open-label assessment of the safety, tolerability, and efficacy of transdermal cannabidiol (ZYN002) for the treatment of pediatric fragile X syndrome

  Median score Mean (SD) score Median change from screening to week 12
Screening (n = 18) Week 12 (n = 18) Screening (n = 18) Week 12 (n = 18) Score (% change) Paired-samples t test
t Cohen’s d
Primary efficacy measures
 ADAMS
  Total 32.00 18.00 32.10 (14.36) 18.10 (8.32) − 15.50 (− 48.90%) − 5.74*** 1.36
  Manic/hyperactive 9.00 6.00 8.80 (3.99) 6.10 (3.29) − 2.50 (− 32.50%) − 4.51*** 1.05
  Depressed mood 2.00 1.00 2.90 (3.94) 2.00 (2.35) 0.00 − 1.54 0.37
  Social avoidance 10.00 5.00 9.90 (5.18) 4.80 (2.07) − 5.50 (− 55.40%) − 4.79*** 1.14
  General anxiety 10.00 4.50 9.40 (4.35) 4.60 (3.35) − 5.00 (− 52.30%) − 7.19*** 1.70
  Compulsive behavior 2.50 1.50 2.70 (2.40) 1.40 (1.42) − 2.00 (− 57.10%) − 2.43* 0.56
Secondary efficacy measures
 ABC-CFXS
  Social avoidance 4.50 2.50 5.10 (3.46) 2.30 (2.22) − 3.00 (− 66.70%) − 4.31*** 1.00
  Stereotypy 8.50 2.00 8.10 (5.91) 3.20 (3.07) − 5.00 (− 69.00%) − 4.20*** 0.99
  Socially unresponsive/lethargic 8.00 4.00 9.20 (6.40) 4.10 (4.09) − 5.00 (− 65.20%) − 3.40** 0.76
  Irritability 18.50 8.50 17.70 (12.68) 10.60 (11.03) − 7.50 (− 50.00%) − 2.92** 0.69
  Hyperactivity 10.50 8.50 13.70 (9.09) 9.80 (7.38) − 3.50 (− 29.80%) − 2.49* 0.59
  Inappropriate speech 6.00 3.50 5.90 (2.30) 3.50 (2.66) − 3.00 (− 44.40%) − 3.69** 0.87
 PARS-R
  Total severity (5-item) 14.50 9.50 15.70 (3.88) 10.60 (3.43) − 6.00 (− 38.20%) − 4.18*** 0.98
 PedsQL
  Total score 65.20 67.80 57.80 (18.78) 67.70 (18.27) 9.80 (16.70%) 2.98* 0.77
  Physical functioning 68.80 79.70 67.90 (27.36) 78.00 (22.39) 6.30 (7.10%) 2.04 0.53
  Emotional functioning 60.00 80.00 64.00 (20.72) 78.30 (16.63) 10.00 (11.10%) 2.27* 0.58
  Social functioning 40.00 50.00 37.30 (24.70) 49.00 (24.35) 10.00 (27.80%) 1.95 0.50
  School functioning 55.00 55.00 55.70 (19.17) 59.10 (22.47) 0.00 0.95 0.25
  Psychosocial health 51.70 62.90 52.40 (17.22) 62.20 (18.91) 8.30 (16.10%) 2.25* 0.58
 VAS
  Hyperactivity/impulsivity 6.70 3.50 5.90 (2.43) 3.60 (2.49) − 1.70 (− 44.60%) − 4.77*** 1.10
  Tantrum/mood lability 5.40 2.90 4.70 (2.09) 3.20 (2.18) − 1.00 (− 33.90%) − 3.59** 0.86
  Anxiety 6.50 3.80 6.00 (2.05) 3.80 (1.93) − 2.20 (− 35.40%) − 4.25*** 1.01
 CGI
  CGI-S 5.00 5.10 (1.39)
  CGI-I 2.00 2.50 (1.01)
  1. Note. Prior to tabulation change in score at week 12 was calculated for each subject by (change = score at week 12 − score at screening), and percent change in score at week 12 was calculated for each subject by [% Change = 100 × (change at week 12/score at screening)]
  2. *p<.05 compared to screening
  3. **p<.01 compared to screening
  4. ***p<.001 compared to screening