- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04669158
Study of Oral Idebenone to Treat Non-Alcoholic Steatohepatitis
October 23, 2023 updated by: Natalie Torok, Stanford University
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.
Study Overview
Status
Active, not recruiting
Conditions
Intervention / Treatment
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 Type
Interventional
Enrollment (Actual)
45
Phase
- Phase 2
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
California
-
Redwood City, California, United States, 94063
- Digestive Health Center, Stanford University
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Stanford, California, United States, 94305
- Stanford University
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
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<10, or based on MRE
Exclusion Criteria:
- 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
- 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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Triple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Idebenone
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.
|
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.
Other Names:
|
Placebo Comparator: Placebo
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.
|
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.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of Participants with Treatment-Related Adverse Events as Assessed by CTCAE v4.0
Time Frame: Week 60: Assessment of AEs
|
Number of Participants with Treatment-Related Adverse Events (Assessed by CTCAE v4.0, including abnormal Physical exams and abnormal laboratory tests results will be reported.
|
Week 60: Assessment of AEs
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in fibrosis stage
Time Frame: Week 48: fibrosis change
|
Number of Participants with change in fibrosis stage
|
Week 48: fibrosis change
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Natalie Torok, MD, Stanford University
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
July 30, 2021
Primary Completion (Estimated)
September 1, 2024
Study Completion (Estimated)
September 1, 2024
Study Registration Dates
First Submitted
December 8, 2020
First Submitted That Met QC Criteria
December 14, 2020
First Posted (Actual)
December 16, 2020
Study Record Updates
Last Update Posted (Actual)
October 25, 2023
Last Update Submitted That Met QC Criteria
October 23, 2023
Last Verified
October 1, 2023
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- 52212
- R21DK111217 (U.S. NIH Grant/Contract)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
UNDECIDED
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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