Table 3 Summary of treatment-emergent AEs
AEs, no. of events, n (%) | Placebo (n = 21) | Volixibat | |||||||
|---|---|---|---|---|---|---|---|---|---|
5 mg b.i.d. (n = 9) | 10 mg q.d. (n = 9) | 20 mg q.d. (n = 9) | 2–5–10–20 mg q.d. (n = 9) | 30 mg q.d. (n = 9) | 40 mg q.d. (n = 9) | 80–40–20 mg q.d. (n = 9) | Total (n = 63) | ||
Any AE | 36, 14 (66.7) | 44, 9 (100) | 35, 9 (100) | 46, 9 (100) | 30, 9 (100) | 21, 9 (100) | 33, 9 (100) | 22, 9 (100) | 231, 63 (100) |
AEs related to study drug | 34, 12 (57.1) | 39, 9 (100) | 32, 9 (100) | 45, 9 (100) | 30, 9 (100) | 18, 9 (100) | 33, 9 (100) | 22, 9 (100) | 219, 63 (100) |
AEs occurring in > 1 participant overall | |||||||||
Gastrointestinal disorders | |||||||||
 Diarrhoeaa | 27, 11 (52.4) | 33, 9 (100) | 27, 9 (100) | 32, 9 (100) | 26, 9 (100) | 16, 9 (100) | 26, 9 (100) | 22, 9 (100) | 182, 63 (100) |
 Anorectal discomfort | 0 | 1, 1 (11.1) | 0 | 0 | 0 | 2, 2 (22.2) | 1, 1 (11.1) | 0 | 4, 4 (6.3) |
 Nausea | 0 | 1, 1 (11.1) | 1, 1 (11.1) | 2, 1 (11.1) | 1, 1 (11.1) | 0 | 0 | 0 | 5, 4 (6.3) |
 Abdominal pain | 1, 1 (4.8) | 0 | 1, 1 (11.1) | 2, 2 (22.2) | 0 | 0 | 0 | 0 | 3, 3 (4.8) |
 Abdominal pain, upper | 0 | 0 | 0 | 3, 3 (33.3) | 0 | 0 | 0 | 0 | 3, 3 (4.8) |
 Gastrointestinal sounds, abnormal | 0 | 0 | 1, 1 (11.1) | 0 | 1, 1 (11.1) | 0 | 1, 1 (11.1) | 0 | 3, 3 (4.8) |
 Vomiting | 0 | 1, 1 (11.1) | 0 | 2, 1 (11.1) | 1, 1 (11.1) | 0 | 0 | 0 | 4, 3 (4.8) |
 Defaecation urgency | 0 | 0 | 0 | 2, 2 (22.2) | 0 | 0 | 0 | 0 | 2, 2 (3.2) |
Nervous system disorders | |||||||||
 Headache | 2, 2 (9.5) | 1, 1 (11.1) | 0 | 1, 1 (11.1) | 1, 1 (11.1) | 0 | 3, 2 (22.2) | 0 | 6, 5 (7.9) |
General disorders and administrative site conditions | |||||||||
 Application site irritation | 1, 1 (4.8) | 0 | 1, 1 (11.1) | 1, 1 (11.1) | 0 | 0 | 0 | 0 | 2, 2 (3.2) |
 Pyrexia | 0 | 2, 2 (22.2) | 0 | 0 | 0 | 0 | 0 | 0 | 2, 2 (3.2) |