Table 1.
Demographics and baseline characteristics (safety analysis set).
| Parameter | Placebo | Tropifexor |
||||
|---|---|---|---|---|---|---|
| 30 μg | 60 μg | 90 μg | 150 μg | |||
| n = 21 | n = 11 | n = 9 | n = 12 | n = 8 | ||
| Age (years), mean (SD) | 53.7 (10.19) | 58.6 (12.42) | 57.9 (11.21) | 53.6 (7.42) | 57.4 (13.81) | |
| Female sex, n (%) | 21 (100.0) | 11 (100.0) | 7 (77.8) | 12 (100.0) | 8 (100.0) | |
| Race, n (%) | ||||||
| Caucasian | 19 (90.5) | 10 (90.9) | 8 (88.9) | 12 (100.0) | 8 (100.0) | |
| Asian/other | 2 (9.5) | 1 (9.1) | 1 (11.1) | – | – | |
| BMI (kg/m2), mean (SD) | 27.3 (5.70) | 26.4 (4.08) | 26.7 (4.91) | 29.1 (7.00) | 26.2 (4.17) | |
| Total daily UDCA dose (mg/kg), mean (SD) | 14.9 (2.64) n = 20∗ |
15.2 (3.21) | 13.7 (3.35) | 14.2 (4.55) n = 11∗ |
16.8 (5.35) n = 7∗ |
|
| History of UDCA use, n (%) | ||||||
| >5 years | 10 (47.6) | 7 (63.6) | 6 (66.7) | 7 (58.3) | 4 (50.0) | |
| >3 to ≤5 years | 2 (9.5) | 2 (18.2) | 1 (11.1) | 3 (25.0) | — | |
| >6 months to ≤3 years | 8 (38.1) | 2 (18.2) | 2 (22.2) | 2 (16.7) | 4 (50.0) | |
| Liver function tests, mean (SD)∗ | Normal range | n = 19 | n = 10 | n = 9 | n = 11 | n = 8 |
| Alkaline phosphatase (U/L) | 35–104 | 338 (122.1) | 316 (173.6) | 303 (96.0) | 306 (92.0) | 284 (109.4) |
| γ-glutamyl transferase (U/L) | 2–65 | 171 (69.2) | 162 (137.1) | 238 (97.9) | 167 (136.2) | 207 (147.6) |
| Alanine aminotransferase (U/L) | 0–45 | 54.2 (22.22) | 55.2 (42.19) | 52.3 (19.75) | 51.5 (22.90) | 54.6 (28.80) |
| Aspartate aminotransferase (U/L) | 0–41 | 48.9 (8.75) n = 16 | 58.6 (39.12) | 57.3 (19.24) | 45.6 (16.98) | 47.3 (20.67) |
| Total bilirubin (μmol/L) | 2–21 | 11.4 (6.64) | 8.2 (4.66) | 11.4 (8.06) | 8.6 (3.32) | 10.4 (4.07) |
| Total PBC-40 QoL score, mean (SD)† | n = 18 | n = 9 | n = 9 | n = 12 | n = 8 | |
| 105.5 (38.33) | 96.1 (31.68) | 91.8 (38.96) | 88.3 (26.50) | 94.8 (42.23) | ||
| VAS itch score (mm), median (min, max)† | n = 20 | n = 9 | n = 9 | n = 12 | n = 8 | |
| 25.0 (0.0, 90.0) | 33.0 (15.0, 67.0) | 35.0 (6.0, 70.0) | 5.0 (0.0, 88.0) | 24.5 (1.0, 86.0) | ||
| VAS itch score ≥40 mm, n (%) | 10 (50.0) | 4 (44.4) | 4 (44.4) | 2 (16.7) | 3 (37.5) | |
QoL, quality of life; UDCA, ursodeoxycholic acid; VAS, visual analogue scale.
∗
One patient was not taking UDCA in the placebo group and data on exact total daily UDCA dose was not available for 1 patient each in the tropifexor 90 and 150 μg groups.
†
Based on pharmacodynamics analysis set.