Table 1.

Ongoing phase 3 trials for patients with NASH

Pharmacologic agents	Mechanism of action	Trial identifier	Expected duration	Patient characteristics	Number	Primary endpoint	Latest results
Obeticholic acid	FXR agonist	REGENERATE (NCT02548351)	2015.9–2022.10	NASH fibrosis (F1-3a)	2,480	1. Improvement of NASH without worsening of fibrosis or improvement of fibrosis without worsening of NASH	Interim 18 month analysis: improvement in fibrosis without worsening of NASH
						2. All-cause mortality and liver-related clinical outcomes
		REVERSE (NCT03439254)	2017.8–2022.6	Compensated cirrhosis due to NASH	540	Percentage of subjects with improvement in fibrosis by at least 1 stage with no worsening of NASH	Not available
Elafibranor	PPAR-α/δ agonist	RESOLVE-IT (NCT02704403)	2016.3–2021.12	NASH fibrosis (NAS score ≥4, F1-3)	2,000	1. Resolution of NASH without worsening of fibrosis	Not available
						2. Composite long-term outcome of all-cause mortality, cirrhosis, and liver-related clinical outcomes
Cenicriviroc	CCR2/CCR5 inhibitor	AURORA (NCT03028740)	2017.4–2028.10	NASH fibrosis (F2-3)	2,000	1. Improvement of fibrosis without worsening of NASH	Not available
						2. Composite long-term outcome of all-cause mortality, cirrhosis, and liver-related clinical outcomes
Resmetirom	Selective THR-β agonist	MAESTRO-NASH (NCT03900429)	2019.3–2024.3	NASH fibrosis (F2-3)	2,000	1. NASH resolution with at least 2 point reduction in NAS and no worsening of fibrosis	Not available
						2. Composite long-term outcome of all-cause mortality, cirrhosis, and liver-related clinical outcomes
Aramchol	SCD1 inhibitor	ARMOR (NCT04104321)	2019.9–2024.12	NASH fibrosis (F2-3)	2,000	1. Resolution of NASH without worsening of fibrosis or improvement of fibrosis without worsening of NASH	Not available
						2. Composite long-term outcome of all-cause mortality, liver transplant, cirrhosis, MELD >15, hospitalization due to hepatic decompensation events
Oltipraz	Liver X receptor-α inhibitor	NCT04142749	2019.11–2021.10	NAFLD (liver fat ≥20% on the MRS)	144	Variation of liver fat by MRS at 24 weeks compared to the baseline (%)	Not available
Dapagliflozin	SGLT2 inhibitor	DEAN (NCT03723252)	2019.3–2021.11	NASH and T2DM (HbA1c <9.5%)	100	Scored liver histological improvement	Not available

NASH, nonalcoholic steatohepatitis; FXR, farnesoid X receptor; PPAR, peroxisome proliferator-activated receptor; NAS, nonalcoholic fatty liver disease activity score; CCR2, C-C chemokine receptor type 2; CCR5, C-C chemokine receptor type 5; THR-β, thyroid hormone receptor beta; SCD1, stearoyl coenzyme A desaturase 1; MELD, model for end-stage liver disease; NAFLD, nonalcoholic fatty liver disease; MRS, magnetic resonance spectroscopy; SGLT2, sodium-glucose cotransporter 2; T2DM, type 2 diabetes mellitus; HbA1c, glycosylated hemoglobin.

^aPatients with stage 1 fibrosis were enrolled only if they have body mass index ≥30, T2DM, or alanine aminotransferase elevation.