Table 3.
TEAEsa Occurring in ≥10% of Patients Who Entered the Extension Phase (Safety Analysis Set).
| TEAE category MedDRA preferred term |
Cohort 1 ≥7 to <12 years (n = 22) |
Cohort 2 ≥2 to <7 years (n = 19) |
Total (N = 41) |
|---|---|---|---|
| Any TEAE, n (%) | 22 (100.0) | 19 (100.0) | 41 (100.0) |
| Pyrexia | 7 (31.8) | 8 (42.1) | 15 (36.6) |
| Upper respiratory tract infection | 6 (27.3) | 5 (26.3) | 11 (26.8) |
| Vomiting | 6 (27.3) | 4 (21.1) | 10 (24.4) |
| Irritabilityb | 5 (22.7) | 3 (15.8) | 8 (19.5) |
| Fatigue | 6 (27.3) | 1 (5.3) | 7 (17.1) |
| Ear infection | 4 (18.2) | 3 (15.8) | 7 (17.1) |
| Lethargy | 3 (13.6) | 4 (21.1) | 7 (17.1) |
| Aggressionb | 2 (9.1) | 5 (26.3) | 7 (17.1) |
| Nasopharyngitis | 3 (13.6) | 3 (15.8) | 6 (14.6) |
| Otitis media | 3 (13.6) | 3 (15.8) | 6 (14.6) |
| Somnolence | 3 (13.6) | 3 (15.8) | 6 (14.6) |
| Cough | 2 (9.1) | 4 (21.1) | 6 (14.6) |
| Abdominal pain, upper | 5 (22.7) | 0 (0.0) | 5 (12.2) |
| Increased appetite | 4 (18.2) | 1 (5.3) | 5 (12.2) |
| Weight increased | 4 (18.2) | 1 (5.3) | 5 (12.2) |
| Headache | 3 (13.6) | 2 (10.5) | 5 (12.2) |
| Dizziness | 2 (9.1) | 3 (15.8) | 5 (12.2) |
Abbreviations: MedDRA, Medical Dictionary for Regulatory Activities; TEAE, treatment-emergent adverse event.
a A TEAE is defined as an adverse event with an onset date, or a worsening in severity from baseline (pretreatment), on or after the first dose of study drug up to 30 days following study drug discontinuation. A patient with 2 or more adverse events in the same preferred term is counted only once for that preferred term.
b There was 1 patient who experienced events of both irritability and aggression; the event of irritability occurred on day 17 of the core study and the event of aggression occurred during the extension phase on day 98 of treatment. The event of aggression led to study discontinuation. There were no other patients who experienced both irritability and aggression.