Table 1.
Demographics and baseline characteristics for pooled LGS and DS populations, overall and on clobazam (intention‐to‐treat population)
| Pooled LGS | Pooled DS | |||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| LGS overall | LGS on clobazam | DS overall | DS on clobazam | |||||||||
| Placebo (n = 161) | CBD10 (n = 73) | CBD20 (n = 162) | Placebo (n = 79) | CBD10 (n = 37) | CBD20 (n = 78) | Placebo (n = 124) | CBD10 a (n = 66) | CBD20 (n = 128) | Placebo (n = 79) | CBD10 (n = 45) | CBD20 (n = 80) | |
| Age, years | ||||||||||||
| Mean | 15.3 | 15.4 | 15.7 | 12.5 | 14.7 | 15.2 | 9.7 | 9.2 | 9.5 | 9.7 | 9.1 | 9.3 |
| SD | 9.5 | 9.5 | 9.7 | 7.5 | 7.8 | 9.3 | 4.7 | 4.3 | 4.5 | 5.0 | 4.1 | 4.3 |
| Sex, n (%) | ||||||||||||
| Male | 87 (54) | 40 (55) | 90 (56) | 42 (53) | 16 (43) | 43 (55) | 58 (47) | 27 (41) | 71 (56) | 35 (44) | 23 (51) | 45 (56) |
| Region, n (%) | ||||||||||||
| USA | 128 (80) | 60 (82) | 121 (75) | 71 (90) | 36 (97) | 70 (90) | 69 (56) | 30 (46) | 66 (52) | 52 (66) | 22 (49) | 44 (55) |
| Rest of world | 33 (21) | 13 (18) | 41 (25) | 8 (10) | 1 (3) | 8 (10) | 55 (44) | 36 (55) | 62 (48) | 27 (34) | 23 (51) | 36 (45) |
| Weight at baseline, kg | ||||||||||||
| Mean | 44.2 | 44.3 | 41.9 | 40.6 | 42.0 | 42.5 | 34.6 | 32.8 | 34.0 | 34.9 | 33.5 | 34.8 |
| SD | 23.0 | 26.2 | 21.6 | 21.9 | 21.0 | 21.4 | 16.5 | 16.6 | 18.0 | 17.5 | 17.3 | 20.0 |
| Body mass index at baseline, kg/m2 | ||||||||||||
| Mean | 20.5 | 20.9 | 20.1 | 19.9 | 20.0 | 21.1 | 18.9 | 18.5 | 18.6 | 19.0 | 18.9 | 19.0 |
| SD | 6.3 | 7.6 | 8.3 | 5.3 | 5.6 | 10.0 | 4.3 | 4.5 | 4.5 | 3.8 | 5.0 | 4.9 |
| Baseline primary seizure frequency, per 28 days b | ||||||||||||
| Median | 79.0 | 86.9 | 78.1 | 80.5 | 87.0 | 64.9 | 15.5 | 13.5 | 10.4 | 17.0 | 13.1 | 10.4 |
| Range | 8.7, 3174.6 | 14.0, 7494.0 | 10.3, 1092.0 | 8.7, 3174.6 | 14.0, 7494.0 | 10.3, 682.3 | 3.0, 770.5 | 0.0, 467.0 | 3.9, 1716.7 | 3.0, 448.9 | 4.0, 238.4 | 3.9, 661.2 |
| Number of AEDs, median (range) | ||||||||||||
| Previous AEDs | 6 (0, 28) | 6 (0, 21) | 6 (1, 18) | 5 (0, 16) | 6 (1, 19) | 5 (1, 18) | 4 (0, 14) | 4 (0, 19) | 4 (0, 26) | 4 (0, 14) | 4 (0, 12) | 4 (0, 20) |
| Current AEDs | 3 (1, 5) | 3 (1, 5) | 3 (0, 5) | 3 (1, 5) | 3 (1, 5) | 3 (1, 5) | 3 (1, 5) | 3 (1, 5) | 3 (1, 5) | 3 (1, 5) | 3 (1, 5) | 3 (1, 5) |
| Most common (>25% in any group) AEDs currently being taken, n (%) | ||||||||||||
| Clobazam | 79 (49) | 37 (51) | 78 (48) | 79 (100) | 37 (100) | 78 (100) | 79 (64) | 45 (68) | 80 (63) | 79 (100) | 45 (100) | 80 (100) |
| Valproate c | 63 (39) | 27 (37) | 64 (40) | 24 (30) | 7 (19) | 22 (28) | 82 (66) | 44 (67) | 84 (66) | 50 (63) | 30 (67) | 56 (70) |
| Levetiracetam | 58 (36) | 22 (30) | 47 (29) | 32 (41) | 9 (24) | 26 (33) | 31 (25) | 19 (29) | 37 (29) | 17 (22) | 11 (24) | 15 (19) |
| Lamotrigine | 56 (35) | 22 (30) | 53 (33) | 22 (28) | 10 (27) | 29 (37) | 5 (4) | 0 | 2 (2) | 3 (4) | 0 | 1 (1) |
| Topiramate | 24 (15) | 13 (18) | 22 (14) | 12 (15) | 4 (11) | 11 (14) | 32 (26) | 11 (17) | 34 (27) | 18 (23) | 8 (18) | 18 (23) |
| Rufinamide | 41 (26) | 19 (26) | 51 (32) | 19 (24) | 12 (32) | 22 (28) | 3 (2) | 2 (3) | 9 (7) | 3 (4) | 1 (2) | 5 (6) |
| Stiripentol | ‐ | ‐ | ‐ | ‐ | ‐ | ‐ | 45 (36) | 25 (38) | 52 (41) | 31 (39) | 17 (38) | 41 (51) |
Abbreviations: AED, antiepileptic drug; CBD10, cannabidiol 10 mg/kg/day; CBD20, cannabidiol 20 mg/kg/day; DS, Dravet syndrome; LGS, Lennox–Gastaut syndrome; SD, standard deviation.
aOne patient randomized to CBD10 was not treated and was withdrawn by the principal investigator.
bBaseline period included all seizure data prior to day 1.
c Valproate includes Ergenyl Chrono for all trials.