Gastroenterology
PHASE2
● Phase II
Phase 1/2a trial tests oral idebenone safety in adults with NASH and fibrosis
ClinicalTrials.gov
Published March 30, 2026
Stanford University
NCT04669158 ↗
This was a randomized, double-blinded, placebo-controlled, single-center phase 1/2a study. It enrolled 53 adults aged 18 years or older with non-alcoholic steatohepatitis (NASH) and stage 1-3 fibrosis. The intervention was oral idebenone, administered at escalating doses starting at 200 mg once daily for 2 weeks, then 200 mg twice daily for 2 weeks, then 200 mg three times daily for up to 48 weeks. The comparator was placebo. The primary outcome was the number of participants with treatment-related adverse events as assessed by CTCAE v4.0. Secondary outcomes included target engagement and fibrosis improvement. The study follow-up duration was not reported. No main results for safety, efficacy, or tolerability were provided in the input data. Adverse events, serious adverse events, discontinuations, and tolerability were all listed as 'not reported.' Key limitations were not specified in the provided information. Funding sources and conflicts of interest were also not reported. The practice relevance was not reported. Given the early-phase nature of the study and the absence of reported safety or efficacy data, no conclusions regarding the clinical utility of idebenone for NASH can be drawn from this summary.
For people living with NASH, a progressive form of fatty liver disease that can lead to scarring and liver failure, treatment options are limited. A new, early-stage study has begun testing whether an oral drug called idebenone might one day help. The study involved 53 adults with NASH and early to moderate liver scarring. They received either the drug or a placebo for up to 48 weeks, starting with a low dose that was gradually increased. The main goal was simply to check for safety and side effects, not to prove the drug works. This is a crucial first step, but it's important to know that the results for safety, side effects, and any potential impact on liver scarring have not been reported yet. This was a small, single-center study, which means we need much larger and longer trials to understand if this drug is truly safe and effective.
What this means for you: Early safety study for a new NASH drug is complete, but results are not yet available.
View Original Abstract ↓
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
