Table 2 Summary of neurological findings and feeding difficulties observed in > 10% of episodes in any treatment group at baseline
Symptoms | Carglumic acid episodes (n = 38) | NH3 scavengers episodes (n = 33) | Carglumic acid + NH3 scavengers episodes (n = 27) | ||||
|---|---|---|---|---|---|---|---|
Baseline n episodes (%) | Endpoint n episodes (%) | Baseline n episodes (%) | Endpoint n episodes (%) | Baseline n episodes (%) | Endpoint n episodes (%) | ||
Neurological findings | Abnormal movements | 10 (26.3) | 1 (2.6) | 3 (9.1) | 1 (3.0) | 5 (18.5) | 1 (3.7) |
Coma | 6 (15.8) | 1 (2.6) | 1 (3.0) | 0 | 3 (11.1) | 2 (7.4) | |
Lethargy | 16 (42.1) | 4 (10.5) | 11 (33.3) | 2 (6.1) | 17 (63.0) | 2 (7.4) | |
Muscle hypotonia | 15 (39.5) | 7 (18.4) | 12 (36.4) | 1 (3.0) | 19 (70.4) | 4 (14.8) | |
Neurological development impairment | 2 (5.3) | 1 (2.6) | 4 (12.1) | 3 (9.1) | 0 | 0 | |
Seizures | 2 (5.3) | 0 | 4 (12.1) | 1 (3.0) | 3 (11.1) | 0 | |
Somnolence/ asthenia | 18 (47.4) | 3 (7.9) | 8 (24.2) | 1 (3.0) | 21 (77.8) | 2 (7.4) | |
Visual impairment | 4 (10.5) | 3 (7.9) | 1 (3.0) | 1 (3.0) | 1 (3.7) | 2 (7.4)a | |
Feeding difficulties | Poor feeding | 15 (39.5) | 4 (10.5) | 13 (39.4) | 0 | 18 (66.7) | 1 (3.7) |
Vomiting | 19 (50.0) | 7 (18.4) | 19 (57.6) | 3 (9.1) | 10 (37.0) | 1 (3.7) | |