Table 2.
Characteristics of the study participants
| Study | Treatment arm | No. of participants | Age, years | Male sex, % | No. of prior ASMs | No. of concomitant ASMs | Baseline monthly convulsive seizures frequency |
|---|---|---|---|---|---|---|---|
|
STICLO–France [19] |
Placebo | 20 | 9.3 (4.9) | 55.0 | 7.5 (2.9) | Valproate and clobazam ongoing drugs | 18.5 (17.0) |
| STP: 50 mg/kg | 21a | 9.4 (4.0) | 28.6 | 6.6 (2.5) | Valproate and clobazam ongoing drugs | 17.9 (17.3) | |
|
STICLO–Italy [20] |
Placebo | 11 | 8.7 (4.4) | 83.3 | – | Valproate and clobazam ongoing drugs | 27.4 (28.6) |
| STP: 50 mg/kg | 12 | 9.2 (3.6) | 66.7 | – | Valproate and clobazam ongoing drugs | 33.6 (28.2) | |
| GWPCARE1A [21] | Placebo | 7 | 7.0 (0.9) | 71.4 | 5 (1–5)b | 2 (1–3)b | – |
| CBD: 5 mg/kg | 10 | 7.2 (1.9) | 50.0 | 3 (0–11)b | 3 (1–4)b | – | |
| CBD: 10 mg/kg | 8 | 7.4 (2.1) | 37.5 | 3.5 (0–5)b | 3 (2–3)b | – | |
| CBD: 20 mg/kg | 9 | 8.7 (1.8) | 33.3 | 4 (2–8)b | 3 (1–4)b | – | |
| GWPCARE1B [22] | Placebo | 59 | 9.8 (4.8) | 45.8 | 4 (0–14)b | 3 (1–5)b | 14.9 (7.0–36.0)c |
| CBD: 20 mg/kg | 61 | 9.7 (4.7) | 57.4 | 4 (0–26)b | 3 (1–5)b | 12.4 (6.2–28.0)c | |
| GWPCARE2 [23] | Placebo | 65 | 9.6 (range 2.2–18.1) | 47.7 | 4 (0–11)b | 3 (1–5)b | 16.6 (7.0–51.1)c |
| CBD: 10 mg/kg | 66 | 9.2 (range 2.3–17.7) | 40.9 | 4 (0–19)b | 3 (1–5)b | 13.5 (6.0–31.2)c | |
| CBD: 20 mg/kg | 67 | 9.3 (range 2.2–18.9) | 53.7 | 4 (0–11)b | 3 (1–4)b | 9.0 (6.3–21.2)c | |
| Lagae et al., 2019 [24] | Placebo | 40 | 9.2 (5.1) | 52.5 | – | 2.5 (0.9) | 27.3 (3.3–147.3)c |
| FFA: 0.2 mg/kg | 39 | 9.0 (4.5) | 56.4 | – | 2.5 (1.1) | 17.5 (4.7–623.5)c | |
| FFA: 0.8 mg/kg | 40 | 8.8 (4.4) | 52.5 | – | 2.3 (0.9) | 20.7 (4.8–124)c | |
| Nabbout et al., 2019 [25] | Placebo | 44 | 9.4 (5.1) | 61.4 | – | 3.4 (0.6) | 10.7 (3–163)c |
| FFA: 0.5 mg/kg | 43 | 8.8 (4.6) | 53.5 | – | 3.7 (0.8) | 14.0 (3–213)c | |
| ELEKTRA [26] | Placebo | 25 | 8.8 (4.5) | 56.0 | – | – | 13.2 (23.9) |
| Soticlestat: ≤ 600 mg | 26 | 8.7 (3.9) | 65.4 | – | – | 13.8 (11.0) |
Data are expressed as mean (SD) unless otherwise specified
ASMs antiseizure medications, CBD pharmaceutical-grade cannabidiol, FFA fenfluramine hydrochloride, IQR interquartile range, SD standard deviation, STP stiripentol
aThe number of patients originally allocated to stiripentol was 22 but one patient was considered not evaluable as they were not compliant
bMedian (range)
cMedian (IQR)