Phase 1/2a trial tests oral idebenone safety in adults with NASH and fibrosis

Status: COMPLETED | Phase: PHASE1/PHASE2 Condition(s): Non Alcoholic Steatohepatitis, Fibrosis Intervention(s): Idebenone (DRUG), Placebo (DRUG) This is a randomized, placebo-controlled phase 1/2a study to investigate the safety and tolerability of Idebenone in patients 18 years of age or older with non-alcoholic steatohepatitis, with stage 1-3 fibrosis. As secondary end point target engagement and fibrosis improvement will be assessed. Detailed: This is a prospective phase 1/2a, randomized, double-blinded, placebo-controlled, single center study of safety and efficacy of oral idebenone in adults 18 years of age or older with non-alcoholic steatohepatitis (NASH). Following IRB approval and written informed consent, 45 participants will be enrolled in the study and randomized in a 1:2 ratio on REDCap data capturing software. Participants will be randomized to two groups and receive the following tablets: placebo, idebenone at escalating doses of 200mg by mouth (P.O.) once a day for 2 weeks, then 200 mg twice a day for 2 weeks, then 3 times per day for the remainder of the study (up to 48 weeks). Monitoring and safety evaluation will continue for 12 weeks after the final dose. The study will investigate the safety and tolerability as Primary Outcome(s): Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0 Enrollment: 53 (ACTUAL) Lead Sponsor: Stanford University Start: 2021-07-30 | Primary Completion: 2023-07-05 Results posted: 2026-03-27