Changes in secondary endpoints over time in treatment responders and nonresponders
| sBA Responsea | sBA and/or Pruritus Responseb | |||||||
|---|---|---|---|---|---|---|---|---|
| Yes | No | Yes | No | |||||
| nc | Mean (SE) | nc | Mean (SE) | nc | Mean (SE) | nc | Mean (SE) | |
| ALT, U/L | ||||||||
| Baseline | 30 | 113 (27) | 51 | 81 (10) | 49 | 105 (18) | 35 | 75 (10) |
| CFB to week 72 | 12 | –141 (63) | 9 | 37 (29) | 16 | –99 (51) | 5 | 44 (49) |
| AST, U/L | ||||||||
| Baseline | 30 | 91 (10) | 51 | 102 (10) | 49 | 100 (9) | 35 | 94 (11) |
| CFB to week 72 | 12 | –59 (14) | 9 | 35 (30) | 16 | –35 (18) | 5 | 33 (46) |
| Total bilirubin, µmol/L | ||||||||
| Baseline | 30 | 28 (6) | 51 | 65 (10) | 49 | 38 (5) | 35 | 68 (14) |
| CFB to week 72 | 12 | –13 (5) | 9 | 5 (10) | 16 | –13 (5) | 5 | 20 (10) |
| Height Z score | ||||||||
| Baseline | 29 | –0.9 (0.2) | 48 | –2.3 (0.2) | 48 | –1.5 (0.2) | 32 | –2.3 (0.3) |
| CFB to week 72 | 12 | 1.0 (0.2) | 10 | 0.6 (0.2) | 16 | 0.9 (0.2) | 6 | 0.6 (0.3) |
| Weight Z score | ||||||||
| Baseline | 30 | –0.3 (0.2) | 48 | –1.5 (0.2) | 49 | –0.7 (0.2) | 32 | –1.5 (0.3) |
| CFB to week 72 | 12 | 0.8 (0.2) | 10 | 0.2 (0.2) | 16 | 0.6 (0.2) | 6 | 0.3 (0.3) |
aPatients with ≥70% reduction in sBAs or sBAs ≤70 µmol/L (baseline level had to be >70 µmol/L for this analysis); bPatients with sBA response and/or pruritus score reduction of ≥1 point from baseline; cPatients with available data at time point for secondary endpoint parameter listed in each row. ALT, alanine aminotransferase; AST, aspartate aminotransferase; CFB, change from baseline; sBA, serum bile acid; SE, standard error.