Phase 2
N=44
Study of Oral Idebenone to Treat Non-Alcoholic Steatohepatitis
Non Alcoholic Steatohepatitis · Fibrosis
Bottom Line
View on ClinicalTrials.gov: NCT04669158 ↗Enrolled (actual)
44
Serious AEs
2.3%
Results posted
Mar 2026
Primary outcome: Primary: Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0 — 9; 4 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Idebenone (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Stanford University
- Primary completion
- Jul 2023
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 |
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.
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
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.