Standard-Dose Ursodeoxycholic Acid Improves Biochemical Liver Function and Fibrosis in Chronic Liver Disease: Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial

Abstract

Background: This study evaluated the efficacy and safety of standard-dose ursodeoxycholic acid (UDCA; fixed daily dose of 300 mg/day) compared with placebo, in patients with chronic liver disease. Methods: A multicenter, randomized, double-blind, placebo-controlled phase IV clinical trial was conducted in academic hospitals in South Korea. Patients with chronic liver disease and abnormal serum alanine aminotransferase (ALT) levels in at least two consecutive results prior to screening, persisting for at least 6 months, were randomly assigned to receive 100 mg UDCA or placebo three times daily for 8 weeks. The primary endpoint was the mean relative change in ALT levels from baseline. The secondary endpoints included changes in fibrosis and drug-related adverse events. Results: A total of 262 patients were analyzed (132 in the UDCA group and 130 in the placebo group). By week 8, there was a significantly greater reduction in serum ALT levels from baseline in the UDCA-treated patients than in the placebo group (-14.70 vs. -5.51 U/L; P = 0.010). The ALT normalization rates were higher in the UDCA group (26.52% vs. 13.08%; odds ratio, 2.60; P = 0.005). Fibrosis reduction, as assessed by the FibroTest score, was greater in the UDCA group (-0.03 vs. -0.00; P = 0.016). The frequency of adverse events in the two groups was similar, with no serious adverse events reported in the UDCA group. Conclusion: In patients with chronic liver disease, 100 mg UDCA three times daily for 8 weeks improved ALT levels and fibrosis, and had a favorable safety profile. Trial Registration: ClinicalTrials.gov Identifier: NCT06272630