Non Alcoholic SteatohepatitisPhase 1/2NCT04669158
Study of Oral Idebenone to Treat Non-Alcoholic Steatohepatitis
A Phase 1/2 study testing Idebenone in people with Non Alcoholic Steatohepatitis and Fibrosis, run by Stanford University at 2 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Non Alcoholic Steatohepatitis, Fibrosis
- What is being tested?
- Idebenone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 53 participants
- Where
- 2 sitesUnited States
- Study dates
- 30 Jul 2021to 5 Jul 2023
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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<10, or based on MRE
Who cannot join
- 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
Show all 22Show fewer
- 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
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Digestive Health Center, Stanford University
Redwood City, California, 94063
Stanford University
Stanford, California, 94305
About this study
Official title: A Pilot Study to Treat Adults With Non-Alcoholic Steatohepatitis With Oral Idebenone
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.
Read the detailed description
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 end point and assess target engagement and fibrosis improvement as secondary end points.
- Conditions
- Non Alcoholic Steatohepatitis, Fibrosis
- Being tested
- Idebenone, Placebo
- Medical subject headings
- Non-alcoholic Fatty Liver Disease, Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IdebenoneDrug
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.
PlaceboDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 60 weeks
Secondary outcomes (1)
Change in Magnetic Resonance Elastography (MRE) as a Measure of Change in Fibrosis Stage
MR elastography is a standard of care technique to assess liver stiffness, that is a highly accurate non-invasive method. MRE creates elastograms (stiffness maps) and data generated in kilopascals (kPa) are correlated to fibrosis stage. Change in stiffness values indicate change in fibrosis. < 2.5 kPa: normal; 2.5 to 3.0 kPa: normal or inflammation; 3.0 to 3.5 kPa: stage 1-2 fibrosis; 3.5 to 4.0 kPa: stage 2-3 fibrosis; 4.0 to 5.0 kPa: stage 3-4 fibrosis; > 5.0 kPa: Stage 4 fibrosis or cirrhosis.
Measured: Week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Dec 2020
- Study start30 Jul 2021
- Primary completion5 Jul 2023
- Study completion5 Jul 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Non Alcoholic SteatohepatitisStudies of the same condition, open and under way.
- More trials run by Stanford UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 53 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Natalie Torok, Stanford University
- Investigators
- Natalie Torok, MD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04669158
- Sponsor's protocol ID
- 52212
- Other IDs
- R21DK111217
- Status last verified
- Mar 2026
- First submitted
- 8 Dec 2020
- Last updated
- 24 Apr 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study to Treat Adults With Non-Alcoholic Steatohepatitis With Oral Idebenone
- ClinicalTrials.gov ID
- NCT04669158
- Acronym / sponsor ID
- 52212
- Conditions
- Non Alcoholic Steatohepatitis, Fibrosis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 53 participants (actual)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Collaborators
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Study start
- 30 Jul 2021 (actual)
- Primary completion
- 5 Jul 2023 (actual)
- Study completion
- 5 Jul 2023 (actual)
- First posted
- 16 Dec 2020 (actual)
- Results
- Posted 27 Mar 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 description
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 end point and assess target engagement and fibrosis improvement as secondary end points.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Idebenone | Experimental | Drug: IdebenoneParticipants 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. |
| Placebo | Placebo comparator | Drug: PlaceboParticipants 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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):
Key exclusion criteria (22 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| IdebenoneIdebenone, 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. | Drug | Idebenone |
| PlaceboPlacebo 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. | Drug | Placebo |
Primary outcome
Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0
Time frame: 60 weeks
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.
1 secondary outcome
Change in Magnetic Resonance Elastography (MRE) as a Measure of Change in Fibrosis Stage
Time frame: Week 48
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Apr 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Natalie Torok, MD principal investigator
Stanford University
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Idebenone in people with Non Alcoholic Steatohepatitis and Fibrosis, run by Stanford University at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 27 Mar 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0 (time frame: 60 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Apr 2026.