Table 1.
Principal characteristics of the study of the drugs in clinical trials.
| Study | Phase | Pt Number | Dose | Study Duration | Primary Endpoint | Primary Endpoint Met | Note | |
|---|---|---|---|---|---|---|---|---|
| Non-Bile Acids FXR agonists (drugs) | ||||||||
| Cilofexor | [32] | 2 | 71 | 30 mg, 100 mg | 12 weeks | Safety and tolerability of Cilofexor | yes | |
| Tropifexor | [33] | 2 | 61 | 30 µg, 60 µg, 90 µg | 12 weeks | Change in GGT in 4 weeks | yes | at interim analysis |
| EDP-305 | NCT03394924 | 2 | 68 | Dose 1 dose 2 | 12 weeks | 20% reduction in ALP or normalization of ALP in 12 weeks | n/a | ongoing |
| PPAR agonists (drugs) | ||||||||
| Seladelpar | [34] | 2 | 70 | 50 mg, 200 mg | 12 weeks | Change in ALP | Early stopped (grade 3 increases in ALT) | |
| [35] | 2 | 5 mg, 10 mg | 12 weeks | Change in ALP | yes | |||
| NCT02955602 | 2 | 119 | 2 mg, 5 mg, 10 mg | 8 weeks with 44 weeks extension | Change in ALP | n/a | ongoing | |
| NCT03602560 (ENHANCE) | 3 | 240 * | 5–10 mg, 10 mg | 52 weeks | Change in ALP and bilirubin | suspended (interface hepatits) | ||
| Elafibranor | [36] | 2 | 45 | 80 mg, 120 mg | 12 weeks | Change in ALP | yes | |
| Fibroblast growth factor 19 (FGF19) analogues (drugs) | ||||||||
| NGM282 | [37] | 2 | 45 | 0.3 mg, 3 mg | 28 days | Change in ALP | yes | |
| Antifibrotic agent (drugs) | ||||||||
| Setanaxib | [38] | 2 | 111 | 400 mg od/bd | 24 weeks | Change in GGT | yes | at interim analysis |
| Immunomodulatory Strategies (drugs) | ||||||||
| Rituximab | [39] | Open label | 6 | 1 g (2 doses) |
52 weeks | Reduction in ALP, IgM and AMA after 36 week | ||
| [40] | Open label | 14 | 1 g (2 doses) |
6 months | Normalization or ALP < 25% from baseline | no | ||
| [41] | 2 | 57 | 1 g (2 doses) |
12 months | Fatigue (PBC 40) | no | ||
| Ustekimumab | [42] | Open label | 20 | 90 mg | 28 weeks | ALP < 40% from baseline | no | |
| Abataceb | [43] | Open label | 16 | 125 mg | 24 weeks | ALP normalization or <40% from baseline | no | |
| Baricitinib | NCT03742973 | 2 | 2 | 2 mg, 4 mg | 12 weeks | Change in ALP | no | Enrollment futility |
| FFP104 | NCT02193360 | 1/2 | 24 (estimated) | 1 mg/kg, 2.5 mg/kg, 2 mg/kg ev | 12 weeks | Safety and tolerability | n/a | Recruitment status unknown |
| E6011 | NCT03092765 | 2 | 29 | High or low dose | 64 weeks | ALP change at week 12 | n/a | Terminated |
| Etrasimod | NCT03155932 | Open label | 2 | 24 weeks | ALP change | n/a | ongoing | |
| Other treatment | ||||||||
| S-adenosyl-L-methionine | [44] | Open label | 24 | 1.2 g | 6 months | PBC 40 improvement | yes | significant decrease of ALP in non-cirrhotic patients |
* estimated.