Study of Oral Idebenone to Treat Non-Alcoholic Steatohepatitis

NCT04669158 | Completed Phase 1 Results FDA Drug

• 2 other identifiers: 52212R21DK111217
• Study Type: interventional
• Target: 53
• Locations: 1 country
• Sites: 2

Brief 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.

Conditions

Fibrosis; Non Alcoholic Steatohepatitis

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0

  Number of Participants with Treatment-Related Adverse Events (Assessed by CTCAE v4.0), and number of participants with abnormal Physical exams and abnormal laboratory tests results are reported.

  60 weeks

Secondary Outcomes (1)

• Change in Magnetic Resonance Elastography (MRE) as a Measure of Change in Fibrosis Stage

  Week 48

Study Arms (2)

Idebenone

EXPERIMENTAL

Participants will take Idebenone 200 mg for 48 weeks, with study visits in clinic for physical and laboratory assessments at weeks 0, 2, 4, 12, 24, 36, and 48 weeks during treatment period and at 4, and 12 weeks after the last dose.

Drug: Idebenone

Placebo

PLACEBO COMPARATOR

Participants will take Placebo for 48 weeks, with study visits in clinic for physical and laboratory assessments at weeks 0, 2, 4, 12, 24, 36, and 48 weeks during treatment period and at 4, and 12 weeks after the last dose.

Drug: Placebo

Interventions

Idebenone DRUG

Idebenone, initially 200mg by mouth (P.O.) once a day for 2 weeks, then 200 mg twice a day for 2 weeks, then 200 mg three times per day for the remainder of the study will be used.

Idebenone

Placebo DRUG

Placebo to match Idebenone once a day for 2 weeks, then twice a day for 2 weeks, then three times per day for the remainder of the study will be used.

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• \) Male or non-pregnant/ non-lactating women ≥ 18 years of age
• \) 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\<10, or based on MRE

You may not qualify if:

• Presence of any other form of liver disease, including viral hepatitis, autoimmune hepatitis, alcoholic liver disease, genetic causes of chronic liver disease):
• Subjects with other etiologies of chronic liver disease, such as chronic hepatitis B and C; autoimmune hepatitis; and inherited liver disease.
• ALT\>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

Sponsors & Collaborators

• Stanford University: lead
• National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK): collaborator

Study Sites (2)

Digestive Health Center, Stanford University

Redwood City, California, 94063, United States

Location

Stanford University

Stanford, California, 94305, United States

Location

MeSH Terms

Conditions

Non-alcoholic Fatty Liver Disease; Fibrosis

Interventions

idebenone

Condition Hierarchy (Ancestors)

Fatty Liver; Liver Diseases; Digestive System Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Natalie Torok, MD
• Organization: Stanford University

ntorok@stanford.edu

(650) 498-7878

Study Officials

• Natalie Torok, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: December 8, 2020
• First Posted: December 16, 2020
• Study Start: July 30, 2021
• Primary Completion: July 5, 2023
• Study Completion: July 5, 2023
• Last Updated: April 24, 2026
• Results First Posted: March 27, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)