Table 2.
Safety summary (safety population)
| Patients, n (%) | Capsule | Tablet | Total | Grade | ||||||
| 100 mg | 200 mg | 400 mg | 600 mg | 800 mg | 200 mg | 300 mg | 400 mg | 3–5 | ||
| (n=1) | (n=3) | (n=7) | (n=30) | (n=7) | (n=3) | (n=3) | (n=30) | (N=84) | (n=84) | |
| Any AE | 1 (100) | 3 (100) | 7 (100) | 30 (100) | 7 (100) | 3 (100) | 3 (100) | 30 (100) | 84 (100) | |
| Related AE | 1 (100) | 2 (66.7) | 7 (100) | 30 (100) | 7 (100) | 3 (100) | 3 (100) | 29 (96.7) | 82 (97.6) | |
| Related SAEs | 0 | 0 | 0 | 8 (26.7) | 1 (14.3) | 0 | 1 (33.3) | 4 (13.3) | 14 (16.7) | |
| AE leading to death | 0 | 0 | 0 | 2 (6.7) | 0 | 0 | 0 | 5 (16.7) | 7 (8.3) | |
| AE leading to discontinuation | 0 | 0 | 1 (14.3) | 17 (56.7) | 3 (42.9) | 0 | 0 | 9 (30.0) | 30 (35.7) | |
| AE leading to treatment interruption | 1 (100) | 0 | 4 (57.1) | 22 (73.3) | 5 (71.4) | 0 | 1 (33.3) | 28 (93.3) | 61 (72.6) | |
| AE leading to dose reduction | 0 | 0 | 1 (14.3) | 1 (3.3) | 0 | 0 | 1 (33.3) | 6 (20.0) | 9 (10.7) | |
| Events occurring in ≥10% of patients | ||||||||||
| Blood bilirubin increased | 0 | 0 | 3 (42.9) | 13 (43.3) | 3 (42.9) | 2 (66.7) | 0 | 15 (50.0) | 36 (42.9) | 20 (23.8) |
| Diarrhoea | 0 | 0 | 1 (14.3) | 17 (56.7) | 4 (57.1) | 0 | 1 (33.3) | 19 (63.3) | 42 (50.0) | 5 (6.0) |
| Hyperbilirubinaemia | 0 | 0 | 2 (28.6) | 10 (33.3) | 4 (57.1) | 0 | 1 (33.3) | 11 (36.7) | 28 (33.3) | 16 (19.0) |
| Stomatitis | 0 | 0 | 1 (14.3) | 11 (36.7) | 4 (57.1) | 1 (33.3) | 0 | 17 (56.7) | 34 (40.5) | 3 (3.6) |
| Palmar-plantar eryrthodysaestesia | 0 | 0 | 2 (28.6) | 11 (36.7) | 1 (14.3) | 0 | 1 (33.3) | 15 (50.0) | 30 (35.7) | 4 (4.8) |
| Arthralgia | 0 | 0 | 1 (14.3) | 11 (36.7) | 4 (57.1) | 1 (33.3) | 2 (66.7) | 9 (30.0) | 28 (33.3) | 2 (2.4) |
| Dry mouth | 0 | 0 | 1 (14.3) | 14 (46.7) | 1 (33.3) | 1 (33.3) | 12 (40.0) | 29 (34.5) | 0 | |
| Fatigue | 0 | 0 | 1 (14.3) | 10 (33.3) | 2 (28.6) | 1 (33.3) | 0 | 9 (30.0) | 23 (27.4) | 5 (6.0) |
| Epistaxis | 0 | 1 (33.3) | 1 (14.3) | 8 (26.7) | 3 (42.9) | 0 | 1 (33.3) | 14 (46.7) | 28 (33.3) | 0 |
| Jaundice | 0 | 0 | 3 (42.9) | 10 (33.3) | 2 (28.6) | 0 | 1 (33.3) | 11 (36.7) | 27 (32.1) | 2 (2.4) |
| Asthenia | 0 | 1 (33.3) | 1 (14.3) | 8 (26.7) | 2 (28.6) | 0 | 3 (100) | 10 (33.3) | 25 (29.8) | 2 (2.4) |
| Decreased appetite | 0 | 0 | 1 (14.3) | 9 (30.0) | 1 (14.3) | 1 (33.3) | 2 (66.7) | 11 (36.7) | 25 (29.8) | 0 |
| Alopecia | 0 | 0 | 1 (14.3) | 8 (26.7) | 5 (71.4) | 1 (33.3) | 1 (33.3) | 10 (33.3) | 26 (31.0) | 1 (1.2) |
| Dysgeusia | 0 | 0 | 2 (28.6) | 5 (16.7) | 1 (14.3) | 1 (33.3) | 1 (33.3) | 10 (33.3) | 20 (23.8) | 0 |
| Hyperphosphataemia | 0 | 0 | 2 (28.6) | 6 (20.0) | 3 (42.9) | 0 | 1 (33.3) | 8 (26.7) | 20 (23.8) | 0 |
| Weight decreased | 0 | 0 | 1 (14.3) | 10 (33.3) | 1 (14.3) | 0 | 2 (66.7) | 5 (16.7) | 19 (22.6) | 1 (1.2) |
| Nasal dryness | 0 | 0 | 1 (14.3) | 5 (16.7) | 0 | 0 | 1 (33.3) | 6 (20.0) | 13 (15.5) | 0 |
| Conjugated bilirubin increased | 0 | 0 | 0 | 3 (10.0) | 0 | 0 | 1 (33.3) | 7 (23.3) | 11 (13.1) | 0 |
| Myalgia | 0 | 0 | 1 (14.3) | 4 (13.3) | 2 (28.6) | 1 (33.3) | 0 | 3 (10.0) | 11 (13.1) | 2 (2.4) |
| Hypertension | 0 | 0 | 0 | 5 (16.7) | 0 | 1 (33.3) | 0 | 8 (26.7) | 14 (16.7) | 3 (3.6) |
| Nausea | 0 | 0 | 0 | 4 (13.3) | 0 | 0 | 0 | 7 (23.3) | 11 (13.1) | 0 |
| Mucosal inflammation | 0 | 0 | 1 (14.3) | 1 (3.3) | 0 | 0 | 1 (33.3) | 7 (23.3) | 10 (11.9) | 0 |
| Vomiting | 0 | 0 | 1 (14.3) | 2 (6.7) | 0 | 0 | 1 (33.3) | 5 (16.7) | 9 (10.7) | 0 |
| Dry skin | 0 | 0 | 0 | 1 (3.3) | 1 (14.3) | 0 | 1 (33.3) | 7 (23.3 | 10 (11.9) | 0 |
| Onycholysis | 0 | 0 | 0 | 6 (20.0) | 2 (28.6) | 0 | 0 | 3 (10.0) | 11 (13.1) | 1 (1.2) |
AE, adverse event; SAE, serious adverse event.