Table 2. Efficacy outcomes.
| Bumetanide 0.5 mg (N=20) | Bumetanide 1.0 mg (N=23) | Bumetanide 2.0 mg (N=22) | Placebo (N=23) | P-value | |
|---|---|---|---|---|---|
| CARS (full analysis set) | |||||
| Screening | |||||
| N | 20 | 23 | 22 | 23 | |
| Mean (s.d.) | 42.45 (4.18) | 41.43 (6.01) | 41.30 (5.44) | 40.41 (4.89) | |
| Day 90 | |||||
| N | 20 | 23 | 22 | 23 | |
| Mean (s.d.) | 37.48 (5.59) | 38.04 (6.13) | 38.14 (6.19) | 38.78 (4.58) | |
| Change | |||||
| Mean (s.d.) | −4.98 (4.33) | −3.09 (3.30) | −3.16 (3.98) | −1.63 (2.34) | |
| Median [range] | −5.00 [−11.0/3.0] | −2.50 [−10.0/1.0] | −2.00 [−12.0/3.0] | −1.00 [−8.5/2.0] | |
| Kruskal–Wallis test | 0.069 | ||||
| Steel–Dwass pair-wise comparisons | |||||
| 0.5 mg b.i.d. vs placebo | 0.049 | ||||
| CARS (completers) | |||||
| Screening | |||||
| N | 20 | 23 | 22 | 23 | |
| Mean (s.d.) | 42.45 (4.18) | 41.13 (6.01) | 41.30 (5.44) | 40.41 (4.89) | |
| Day 90 | |||||
| N | 20 | 19 | 13 | 21 | |
| Mean (s.d.) | 37.48 (5.59) | 37.00 (5.31) | 37.73 (7.14) | 38.62 (4.60) | |
| Change | |||||
| Mean (s.d.) | −4.98 (4.33) | −3.74 (3.28) | −5.35 (3.88) | −1.79 (2.39) | |
| Median [range] | −5.00 [−11.0/3.0] | −4.00 [−10.0/1.0] | −6.00 [−12.0/3.0] | −1.00 [−8.5/2.0] | |
| Kruskal–Wallis test | 0.015 | ||||
| Steel–Dwass pair-wise comparisons | |||||
| 2.0 mg b.i.d. vs placebo | 0.017 | ||||
| SRS (total score) | |||||
| Screening | |||||
| N | 20 | 23 | 22 | 22 | |
| Mean (s.d.) | 113.35 (17.48) | 106.42 (23.95) | 106.58 (25.09) | 112.68 (20.65) | |
| Day 90 | |||||
| N | 20 | 18 | 13 | 21 | |
| Mean (s.d.) | 100.99 (23.70) | 87.96 (19.89) | 87.03 (22.03) | 109.47 (26.07) | |
| Change | |||||
| Mean (s.d.) | −12.36 (23.57) | −13.17 (20.45) | −21.83 (19.78) | −1.55 (20.38) | |
| Median [range] | −13.00 [−86.0/14.0] | −6.00 [−52.0/18.0] | −27.00 [−47.0/8.0] | 3.5 [−68.0/27.0] | |
| Kruskal–Wallis test | 0.020 | ||||
| CGI-I | |||||
| CGI-I at day 90 | |||||
| Very much improved | 0 | 0 | 1 (7.7%) | 0 | |
| Much improved | 7 (35.0%) | 5 (26.3%) | 5 (38.5%) | 1 (4.8%) | |
| Minimally improved | 7 (35.0%) | 10 (52.6%) | 7 (53.8%) | 10 (47.6%) | |
| No change | 4 (20.0%) | 4 (21.1%) | 0 | 9 (42.9%) | |
| Minimally worse | 1 (5.0%) | 0 | 0 | 1 (4.8%) | |
| Not assessed/missing | 0 | 4 | 9 | 2 | |
| Kruskal–Wallis test | 0.004 | ||||
Abbreviations: CARS, Childhood Autism Rating Scale; CGI-I, Clinical Global Impressions Improvement scale; SRS, Social Responsive Scale.
Efficacy outcomes, N is number of patients. For CARS and SRS scales, change from screening to day 90. A reduction in CARS and SRS score corresponds to an improvement.