Table 3.
Treatment-emergent adverse events
| Carglumic acid (n = 38) | NH3 scavengers (n = 33) | Carglumic acid + NH3 scavengers (n = 27) | |
|---|---|---|---|
| Number of TEAEs | 54 | 85 | 18 |
| Number of drug-relateda TEAEs | 18 | 1 | 6 |
| Number of serious TEAEs | 13 | 31 | 7 |
| Number of drug-relatedb serious TEAEs | 5 | 0 | 1 |
| TEAEs leading to death | 3 | 13 | 6 |
| TEAEs occurring in ≥10% of episodes in any treatment group, n episodes (%) | |||
| Coagulopathy | 1 (2.6)b | 5 (15.2) | 0 |
| Thrombocytopenia | 2 (5.3)b | 7 (21.2) | 1 (3.7) |
| Hyperglycaemia | 1 (2.6) | 5 (15.2) | 1 (3.7) |
| Hypocalcaemia | 1 (2.6)b | 4 (12.1) | 0 |
| Hypokalaemia | 1 (2.6) | 6 (18.2) | 0 |
| Respiratory distress | 0 | 4 (12.1) | 0 |
NH3 Ammonia, TEAE Treatment-emergent adverse event
aDrug-relatedness in the combination group refers exclusively to carglumic acid treatment and not NH3 scavenger treatment
bConsidered to be drug-related in one episode/patient. n represents the number of episodes evaluated