Table 1.
Treatment‐Related Adverse Events: Study 1—Stage 1 and Stage 2
| Study 1, Single Dose | ||||||||
| Stage 1 | Stage 2 | |||||||
| Fasted | Fed | Fasted | ||||||
| PTC299 Dose, mg/kg | PTC299 Dose, mg/kg | |||||||
| Placebo | 0.03 | 0.1 | 0.3 | 1.0 | 3.0 | 1.0 | 1.0 | |
| n = 10 | n = 6 | n = 6 | n = 6 | n = 6 | n = 6 | n = 12 | n = 12 | |
| Number (%) of Subjects With Adverse Event | ||||||||
| Gastrointestinal disorders | ||||||||
| Diarrhea | — | — | — | — | — | 1 (17) | — | 1— (8)a |
| Epigastric discomfort | — | 1 (17) | — | — | — | — | — | — |
| Nausea | — | — | 1 (17) | — | — | — | — | 1 (8) |
| Stomach discomfort | — | 1 (17) | — | — | — | 2 (33) | — | — |
| Vomiting | — | — | — | — | 1 (17) | — | — | — |
| General disorders | ||||||||
| Fatigue | — | — | — | — | — | 1 (17) | — | 1 (8) |
| Feeling hot | — | 2 (33) | — | — | — | — | — | — |
| Nervous system disorders | ||||||||
| Dizziness | — | 1 (17) | — | — | — | — | — | — |
| Headache | — | 1 (17) | — | — | 1 (17) | 1 (17) | — | — |
| Study 2, Multiple Dose | ||||||||
| Stage 1 | Stage 2 | |||||||
| PTC299 mg/kg/dose Twice Daily | Placebo n = 2 | PTC299 mg/kg/ dose 3 Times Daily | ||||||
| 0.3 n = 6 | 0.6 n = 6 | 1.2 n = 6 | 1.6 n = 6 | |||||
| Gastrointestinal disorders | ||||||||
| Diarrhea | 1a (17) | — | — | — | 1 (50) | 2 (33) | ||
aGrade 2 (moderate) event; all other events were grade 1 (mild).