Table 2.
Mean (SD) of clinical outcomes at baseline and day 14 by treatment group and between group differences at day 14 adjusted for baseline
| Placebo | 10 mg | 100 mg | Comparison (mean (95% CI)) of change between treatment groups | ||||||
|---|---|---|---|---|---|---|---|---|---|
| Baseline | Day 14 | Baseline | Day 14 | Baseline | Day14 | 10 mg vs. placebo | 100 mg vs. placebo | Joint test P value | |
| UHDRS – TMS | 32.11 (11.55) | 31.63 (12.07) | 29.53 (14.5) | 26.12 (14.68) | 35.16 (15.37) | 33.05 (10.74) | −3.45 (−7.09, −0.95)† | −1.03 (−5.97, 1.31)† | <0.05 |
| UHDRS – TFC | 10 (2.13) | 9.95 (2.2) | 10.65 (2.06) | 10.65 (2.06) | 9.89 (2.23) | 9.84 (2.24) | 0.06 (−0.07, 0.26)† | −0.0005 (−0.2917, 0.3124)† | >0.05 |
| Functional assessment | 20.37 (3.65) | 20.32 (3.68) | 21.53 (3.45) | 21.41 (3.78) | 20.26 (3.78) | 20.47 (3.47) | −0.04 (−0.5, 0.34)† | 0.26 (−0.15, 0.8)† | >0.05 |
| Independence scale | 83.68 (10.12) | 83.95 (9.8) | 86.47 (11.56) | 85.88 (11.89) | 82.37 (12.18) | 83.16 (11.57) | −0.77 (−2.06, −0.01)† | 0.49 (−0.44, 1.43)† | >0.05 |
| SDMT | 19.61 (7.77) | 21.82 (10.18) | 28.82 (15.61) | 32.29 (13.75) | 21.26 (9.18) | 22.79 (10.76) | 2.04 (−1.94, 6.02) | −0.61 (−4.29, 3.07) | 0.368 |
| Letter fluency | 17.63 (10.59) | 18.37 (10.78) | 23.71 (10.59) | 26.59 (15.66) | 16.53 (8.42) | 20.21 (8.51) | 2.27 (−2.51, 7.05) | 2.92 (−1.58, 7.43) | 0.402 |
| Category fluency | 11.58 (5.4) | 11.74 (4.56) | 13 (6.27) | 15.35 (6.92) | 11 (5.19) | 11.95 (4.64) | 2.52 (0.31, 4.73) | 0.66 (−1.48, 2.8) | 0.073 |
| Stroop colour | 38.32 (13.22) | 40 (13.79) | 50.47 (18.47) | 51.82 (19.56) | 40.11 (15.47) | 39.84 (13.91) | 1.7 (−4.59, 8) | −1.65 (−7.48, 4.18) | 0.559 |
| Stroop word | 56 (16.29) | 54.17 (20.44) | 68.06 (23.46) | 68.06 (22.55) | 56.95 (24.69) | 53.37 (18.85) | 3.98 (−2.33, 10.29) | −1.32 (−7.31, 4.68) | 0.223 |
| Stroop interference | 18.84 (10.37) | 21.79 (11.71) | 28.06 (10.79) | 30.47 (10.38) | 21.26 (9.9) | 23.74 (9.64) | 1.18 (−3.49, 5.85) | −0.02 (−4.3, 4.26) | 0.843 |
| Trail A time | 91.89 (52.67) | 96.22 (65.65) | 70.24 (34.23) | 55.18 (36.87) | 85.68 (61.34) | 84.74 (62.62) | −19.42 (−40.76, 0.15)† | −4.05 (−26.33, 16.13)† | >0.05 |
| Trail B time | 196.65 (62.05) | 191.41 (68.25) | 162.06 (60.73) | 141.41 (72.35) | 179.94 (52.64) | 173.95 (64.27) | −21.11 (−53.18, 5.9)† | −5.14 (−34.05, 24.19)† | >0.05 |
| PSQI | 5.47 (3.45) | 4.83 (3.63) | 5.19 (3.54) | 4.82 (3.52) | 6.11 (4.18) | 5.56 (4.23) | 0.16 (−1.05, 1.38) | 0.69 (−0.5, 1.87) | 0.477 |
| PBA-s mood* | 1.63 (3.11) | 2.26 (3.62) | 2.76 (3.67) | 1.82 (2.86) | 3.11 (5.45) | 2.11 (3.94) | −1.17 (−2.84, 0.39)† | −1.11 (−2.55, 0.17)† | >0.05 |
| PBA-s apathy* | 3.95 (4.14) | 4.11 (4.37) | 2.88 (3.64) | 2.59 (4.15) | 4.32 (4.11) | 3.58 (4.91) | −0.54 (−1.69, 0.91)† | −0.86 (−2.66, 1.14)† | >0.05 |
| PBA-s irritability/aggression* | 1.0 (1.63) | 1.58 (3.02) | 2.47 (3.14) | 1.94 (3.25) | 1.53 (2.86) | 1.0 (3.46) | −0.96 (−2.41, 0.73)† | −1.05 (−2.49, −0.1)† | >0.05 |
| HADS-SIS Anxiety subscore | 4.47 (3.47) | 4.11 (3.26) | 5.29 (4.78) | 4.41 (4.29) | 4.26 (3.28) | 4.63 (4.73) | −0.29 (−2.34, 1.76) | 0.68 (−1.31, 2.67) | 0.619 |
| HADS-SIS Depression subscore | 4.95 (4.58) | 4.95 (4.66) | 5.76 (4.64) | 4.82 (3.61) | 5.21 (3.69) | 5.26 (5.13) | −0.75 (−2.86, 1.36) | 0.11 (−1.93, 2.16) | 0.677 |
| HADS-SIS Irritability subscore | 4.58 (3.99) | 3.53 (3.78) | 6.65 (5.21) | 5.53 (5.34) | 3.95 (3.41) | 4.16 (3.06) | 0.62 (−1.45, 2.68) | 1.06 (−0.91, 3.02) | 0.56 |
| FrSBe-p total score | 76.61 (50.08) | 76.84 (56.81) | 97.47 (86.53) | 93.18 (74.89) | 86.58 (60.4) | 97.37 (67.57) | −0.04 (−19.95, 21.05)† | 13.9 (−3.47, 31.98)† | >0.05 |
| FRsBe executive dysfunction | 28.79 (19.55) | 28.32 (25.95) | 34.18 (33.06) | 33.59 (29.12) | 31.37 (24.24) | 39.47 (30.63) | 0.21 (−9.87, 10.5)† | 8.74 (0.83, 18.66)† | >0.05 |
| FRsBe disinihbition | 16.74 (8.81) | 16.32 (11.4) | 24.35 (22.88) | 22.41 (15.52) | 19.53 (12.73) | 20.21 (13.33) | 0.88 (−5.18, 6.07)† | 1.99 (−2.77, 6.66)† | >0.05 |
| FRsBe apathy | 32.83 (27.06) | 32.21 (28.25) | 38.94 (36.13) | 37.18 (32.57) | 35.68 (30.04) | 37.68 (33.04) | 0.09 (−10.56, 9.34)† | 3.46 (−5.91, 14.89)† | >0.05 |
For the PBA-s, only the three important components mood (depression + suicidal ideation), apathy and a composite of irritability/aggression were evaluated. †Indicates 95% bias corrected and accelerated bootstrap confidence interval instead of normal based confidence interval. Where bootstrap confidence intervals used, the joint test P value is recorded as <0.05 if either of the comparisons with placebo reached statistical significance below P = 0.025, based on assessment of 97.5% confidence intervals. In all other cases, this is the P value from the corresponding joint Wald test F value. Comparisons between 10 mg and 100 mg were not assessed as comparisons with placebo were deemed most relevant. FrSBe-p, frontal systems behavioural scale - participant self-rating; HADS, hospital anxiety and depression scale; PBA-s, problem behaviours assessment - short version; PSQI, Pittsburgh sleep quality index; SDMT, symbol digit modalities test; SIS, Snaith irritability scale; TMS, total motor score; UHDRS, Unified Huntington's Disease Rating Scale.