Table 3.
Efficacy Results for Secondary Outcomes
| Parameter | Pitolisant (n = 201) | Placebo (n = 67) | P Value |
|---|---|---|---|
| OSLER test | |||
| OSLER test mean sleep latency at inclusion, min, (SD) | 14.79 (10.95) | 15.92 (11.04) | — |
| Number of patients with OSLER test = 40 min at inclusion | 11 (5.5%) | 4 (6%) | — |
| Number of patients with OSLER test ≥30 min and <40 min at inclusion | 13 (6.5%) | 3 (4.5%) | — |
| OSLER test mean sleep latency at end of treatment, min | 21.95 (13.53) | 20.25 (13.42) | — |
| Ratio of OSLER test V6/OSLER test V2, geometric mean | 1.65 | 1.39 | 0.120 |
| Mean difference of pitolisant and placebo logarithms of sleep latency at end of DB treatment (95% CI) | 0.1 (0.0–0.3) |
— | |
| Normal vigilance (number of 3–6 and ≥7 errors = 0 for each of the three tests) | |||
| At baseline (V2) | 2.0% (0.5–5.0%) | 3.0% (0.4–10.4%) | — |
| At the end of DB treatment (V6) | 8.5% (4.9–13.5%) | 6.3% (1.7–15.2%) | 0.487 |
| Pichot fatigue score, mean change (SD) | −3.6 (5.6) | −1.0 (6.3) | 0.005 |
| Sleep diary variables | |||
| Mean change in daily number of sleep/sleepiness episodes (SD) | −1.79 (1.97) | −1.30 (1.86) | 0.056* |
| Mean change in daily duration of sleep/sleepiness episodes (SD) | −47.87 (53.39) | −32.24 (48.82) | 0.066† |
| EQ-5D, mean change in VAS score | 7.3 ± 16.2 | 1.8 ± 16.3 | 0.059 |
| Leeds Sleep Evaluation Questionnaire | |||
| Mean change in modified getting to sleep (SD) | 10.21 (24.99) | 2.42 (23.51) | 0.155 |
| Mean change in quality of sleep (SD) | 17.70 (26.08) | 13.00 (25.56) | 0.108 |
| Mean change in awake after sleep (SD) | 19.19 (26.61) | 14.00 (25.18) | 0.160 |
| Mean change in behavior after awakening (SD) | 21.96 (22.26) | 13.35 (20.89) | 0.018 |
| Mean change in global LSEQ score (SD) | 17.26 (14.80) | 10.69 (14.80) | 0.005 |
| TMT A, mean change in average time (SD) | −8.9 (12.7) | −7.3 (13.7) | 0.389 |
| TMT B, mean change in average time (SD) | −22.5 (40.0) | −16.3 (33.8) | 0.648 |
| CGI | <0.001 | ||
| Very much improved | 21 (11.1%) | 3 (4.7%) | |
| Much improved | 84 (44.2%) | 19 (29.7%) | |
| Minimally improved | 55 (28.9%) | 14 (21.9%) | |
| No change | 30 (15.8%) | 22 (34.4%) | |
| Minimally worse | 0 (0.0%) | 6 (9.4%) | |
| Much worse | 0 (0.0%) | 0 (0.0%) | |
| Very much worse | 0 (0.0%) | 0 (0.0%) | |
| CGI improvement at end of DB treatment (V6) | |||
| n (%) | 160 (84.2%) | 36 (56.3%) | |
| 95% CI | 78.2–89.1% | 43.3–68.6% | |
| Patient’s global opinion | <0.001 | ||
| Improvement at V6, n (%) | 164 (86.3%) | 39 (60.9%) | |
| 95% CI | 80.6–90.9% | 47.9–72.9% | |
Definition of abbreviations: CGI = Clinical Global Impression; CI = confidence interval; DB = double-blind; EQ-5D = EuroQol five-dimension quality of life scale; LSEQ = Leeds Sleep Evaluation Questionnaire; OSLER = Oxford Sleep Resistance test; TMT = Trail Making Test; V2 = visit 2; V6 = visit 6; VAS = visual analogue scale.
*
P = 0.049 in the per-protocol population.
†
P = 0.050 in the per-protocol population.