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) | |
Full analysis set. N (%) based on the number of episodes
aFor one patient, neurological status at baseline was erroneously marked as normal despite the patient having an ongoing history of blindness recorded at screening. The patient’s neurological status at endpoint correctly identified visual impairment. NH3 data was censored at extracorporeal detoxification initiation
NH3 ammonia