Phase 2 Completed N=44 Randomized Triple-blind Treatment

Study of Oral Idebenone to Treat Non-Alcoholic Steatohepatitis

Metabolic Dysfunction-Associated Steatohepatitis · Fibrosis

Source: ClinicalTrials.gov NCT04669158 ↗

Enrolled (actual)

Serious AEs

2.3%

Results posted

Mar 2026

Primary outcomePrimary: Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0 — 9; 4 Participants

Summary

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.

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0	9; 4	—
SECONDARY Change in Magnetic Resonance Elastography (MRE) as a Measure of Change in Fibrosis Stage	-1.47; 0.833	0.236

Eligibility Criteria

Inclusion Criteria

1) Male or non-pregnant/ non-lactating women ≥ 18 years of age
2) Diagnosis of NASH: NAFLD activity score (NAS) of 4 or greater with a score of 1 for each of the following (steatosis scored 0-3, lobular inflammation scored 0-3, ballooning scored 0-2):
Steatosis
Lobular inflammation
Hepatocyte ballooning 3) Fibrosis (Ishak fibrosis score ≥1 on liver biopsy) within 6 months of enrollment with MELD 300 U/l
Total serum bilirubin ≥ to 1.3 mg/dL (Gilbert's Syndrome patients excepted)
International Normalized Ratio (INR) ≥ 1.3
MELD>10
Serum creatinine >2.0mg/dl
Known alcohol abuse or alcohol use disorder:
>20 g/day for women
>30 g/day for men
Active substance abuse
Any medical condition that prevents MRE, MR-PDFF
Platelet count ≤100//mm3
Decompensated cirrhosis
Hemoglobin <11 g/dl in females or <12 g/dl in males
Presence/history of HCC
History of liver transplantation
History of bariatric surgery
History of inflammatory bowel disease
History of cardiovascular disease, long QT syndrome.
Subjects who have participated in investigational drug trials and took any investigational drugs within 60 days prior to the first dose of Idebenone. History of receiving other investigations drug within 30 days prior to enrollment
Any concerns regarding compliance by enrolling physician

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Data sourced from ClinicalTrials.gov (NCT04669158). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.