Table 3.
Clinical results from the phase III odevixibat trial.37
| Placebo (n = 20) | Odevixibat 40 μg/kg/day (n = 23) | Odevixibat 120 μg/kg/day (n = 19) | Odevixibat, all doses (n = 42) | |
|---|---|---|---|---|
| Serum bile acid response (%) | 0% | 43% | 21% | 33% |
| Proportion of positive pruritus assessments (%) | 30% | 58% | 52% | 55% |
| Mean change from baseline to week 24 in ObsRO scratching score | -0.25 | NR | NR | -1.11 |
| Most common adverse events (%) | Pyrexia (25%) | Diarrhoea/frequent bowel movements (29%) | Pyrexia (26%) and upper respiratory tract infection (26%) | Diarrhoea/frequent bowel movements (31%) |
ObsRO, observer reported outcome; NR, not reported. The phase III randomised-controlled trial recruited 62 patients with either PFIC1 or PFIC2 (median age, 3.2 years) who received placebo (n = 20), or odevixibat at either 40 μg/kg/day (n = 23) or 120 μg/kg/day (n = 19) for 24 weeks.