TABLE 4.
Summary of treatment-emergent adverse events (safety analysis set)a
| Event | Data for i.v. cohort: |
Data for oral cohort: |
|||||
|---|---|---|---|---|---|---|---|
| 1 to <6 yrs (n = 9) | 6 to <12 yrs (n = 8) | 12 to <18 yrs (n = 10) | Total (n = 27) | 6 to <12 yrs (n = 9) | 12 to <18 yrs (n = 10) | Total (n = 19) | |
| Any TEAE (n [%]) | 7 (77.8) | 8 (100.0) | 10 (100.0) | 25 (92.6) | 9 (100.0) | 9 (90.0) | 18 (94.7) |
| Any serious TEAE (n [%])b | 3 (33.3) | 4 (50.0) | 5 (50.0) | 12 (44.4) | 5 (55.6) | 3 (30.0) | 8 (42.1) |
| Any TEAE leading to discontinuation of study drug | 0 | 1 (12.5) | 1 (10.0) | 2 (7.4) | 3 (33.3) | 1 (10.0) | 4 (21.1) |
| Any drug-related TEAE (n [%])c | 3 (33.3) | 3 (37.5) | 4 (40.0) | 10 (37.0) | 4 (44.4) | 6 (60.0) | 10 (52.6) |
| Any drug-related serious TEAE (n [%])b,c | 0 | 0 | 1 (10.0)d | 1 (3.7) | 1 (11.1)e | 0 | 1 (5.3) |
| Any drug-related TEAE leading to discontinuation of study drug (n [%])c | 0 | 1 (12.5)f | 1 (10.0)d | 2 (7.4) | 3 (33.3)e,g,h | 0 | 3 (15.8) |
| TEAEs occurring in ≥35% of patients in any treatment cohort (n [%]) | |||||||
| Pyrexia | 4 (44.4) | 5 (62.5) | 5 (50.0) | 14 (51.9) | 5 (55.6) | 2 (20.0) | 7 (36.8) |
| Mucosal inflammation | 5 (55.6) | 4 (50.0) | 3 (30.0) | 12 (44.4) | 0 | 1 (10.0) | 1 (5.3) |
| Diarrhea | 2 (22.2) | 4 (50.0) | 3 (30.0) | 9 (33.3) | 2 (22.2) | 1 (10.0) | 3 (15.8) |
| Vomiting | 2 (22.2) | 0 | 0 | 2 (7.4) | 3 (33.3) | 4 (40.0) | 7 (36.8) |
| Pain in extremity | 1 (11.1) | 1 (12.5) | 4 (40.0) | 6 (22.2) | 1 (11.1) | 1 (10.0) | 2 (10.5) |
| Dysuria | 0 | 3 (37.5) | 1 (10.0) | 4 (14.8) | 0 | 0 | 0 |
Safety analysis set included all patients who received ≥1 dose of the study drug. i.v., intravenous; TEAE, treatment-emergent adverse event.
Includes serious TEAEs upgraded by the sponsor based on review of the sponsor’s list of Always Serious terms.
Reasonable possibility that the event may have been caused by the study drug as assessed by the investigator; if relationship was missing, the event was considered drug-related.
Prolonged QT interval in a 16-year-old girl (day –3 [baseline], QTc = 479 msec; day 7, QTc = 484 msec).
Four serious TEAEs (tachycardia, nausea, vomiting, and pyrexia) occurred in a single patient; three of these (nausea, vomiting, and pyrexia) resulted in discontinuation of the study drug.
Increased hepatic enzymes (reported as severe).
Increased transaminases.
Upper abdominal pain.