NCT00501592 | Type 2 diabetes and presumed NAFLD | INT-747 (Obeticholic acid) 25 or 50 mg/d × 6 weeks; oral | Well-tolerated, increased insulin sensitivity, reduced markers of liver inflammation, and fibrosis in patients with type 2 diabetes mellitus and NAFLD (Mudaliar et al., 2013) |
NCT01265498 FLINT trial | NASH | Obeticholic acid 25 mg/d × 72 weeks; oral | Weight loss in up to 44% of patients with NASH; beneficial effects on serum aminotransferases and histology (Hameed et al., 2018) |
NCT01654731 BEZURSO Trial | PBC | Benzafibrate 400 mg/d × 24 months; oral as an adjuvant therapy in patients showing incomplete biochemical response to UDCA treatment (13–15 mg/kg/day) | Complete biochemical response that was significantly higher than that with placebo and UDCA therapy (Corpechot et al., 2018) |
NCT01755507 | PSC | norUDCA 500, 1000, or 1500 mg/d × 12 weeks; oral | norUDCA significantly reduced alkaline phosphatase values dose-dependently in all treatment arms. The safety profile of norUDCA was excellent and comparable to placebo (Fickert et al., 2017) |
NCT02177136 | PSC | Obeticholic acid 1.5 mg titrating to 3 mg or 5 mg titrating to 10 mg/d × 24 weeks; oral | Obeticholic acid 5–10 mg reduced serum ALP in patients with PSC. Mild to moderate dose-related pruritus was the most common adverse event (Kowdley et al., 2020) |
NCT02443116 | NASH | Aldafermin/NGM282 (FGF19 analog) | Reduced liver fat, fibrosis improvement, markedly reduced major hydrophobic bile acids that have greater detergent activity and cytotoxicity. (Harrison et al., 2018: NCT02443116) (Hirschfield et al., 2019: NCT02704364) (Sanyal et al., 2021: NCT02443116, NCT02704364) (Harrison et al., 2021: NCT02443116) |
NCT02704364 | PSC | 0.3, 1, 3, or 6 mg/d × 12 or 24 weeks; subcutaneous |
NCT02548351 REGENERATE Trial (Phase III Trial) | NASH with Fibrosis | Obeticholic acid 10 or 25 mg/d; oral Ongoing. Estimated completion date September 2025. | Interim analysis: Obeticholic acid 25 mg significantly improved fibrosis and key components of NASH disease activity among patients with NASH (Younossi et al., 2019) |