Table 1. Demographic and Baseline Characteristics.
| Characteristic | Participants, No. (%) |
|---|---|
| Safety cohort (48 participants) | |
| Age, mean (SD), y | 10.5 (3.8) |
| Sex | |
| Boys | 26 (54.2) |
| Girls | 22 (45.8) |
| Weight, mean (SD), kg | 39.3 (20.0) |
| Height, mean (SD), cm | 139.0 (20.4) |
| BMI, mean (SD) | 19.2 (4.9) |
| Etiology | |
| Genetic or presumed genetic | 43 (89.6) |
| Nonstructural | 38 (79.2) |
| Structural malformationa | 5 (10.4) |
| Brain injuryb | 5 (10.3) |
| Syndrome | |
| Dravet syndrome | 8 (16.7) |
| Lennox-Gastaut syndrome | 5 (10.4) |
| Myoclonic-atonic epilepsy | 6 (12.5) |
| West syndrome | 3 (6.3) |
| Otherc | 26 (54) |
| Receiving prior medications | |
| Sodium valproate | 34 (70.8) |
| Clobazam | 25 (52.1) |
| Levetiracetam | 17 (35.4) |
| Lamotrigine | 16 (33.3) |
| Topiramate | 13 (27.1) |
| Focal impaired awareness/tonic-clonic seizure cohort (33 participants) | |
| Seizure types analyzedd | |
| Focal impaired awareness seizures | 26 (79) |
| Focal to bilateral tonic-clonic seizures | 7 (21) |
| Generalized tonic-clonic seizures | 14 (42) |
| Median baseline seizure frequency (range) | |
| Focal impaired awareness seizures | 6.22 (0-712.6) |
| Focal to bilateral tonic-clonic seizures | 0 (0-236.4) |
| Generalized tonic-clonic seizures | 0 (0-24) |
Abbreviations: BMI, body mass index (calculated as weight in kilograms divided by height in meters squared); DEE, developmental and epileptic encephalopathies.
a
Includes polymicrogyria, hypothalamic hamartoma, cortical dysplasia, and absence of septum pellucidum.
b
Includes ischemic stroke, hypoxic-ischemic encephalopathy, and traumatic brain injury.
c
Includes generalized epileptic encephalopathy, focal DEE, multifocal DEE, late-onset infantile spasms, and DEE unclassified.
d
During the 1-month baseline period. Patients can appear in more than 1 seizure type and therefore will not sum to 33.