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Study of Oral Idebenone to Treat Non-Alcoholic Steatohepatitis

Primary Purpose

Non Alcoholic Steatohepatitis

Status
Active
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Idebenone
Placebo
Sponsored by
Stanford University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Non Alcoholic Steatohepatitis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Digestive Health Center, Stanford University
  • Stanford University

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Idebenone

Placebo

Arm Description

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.

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.

Outcomes

Primary Outcome Measures

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, including abnormal Physical exams and abnormal laboratory tests results will be reported.

Secondary Outcome Measures

Change in fibrosis stage
Number of Participants with change in fibrosis stage

Full Information

First Posted
December 8, 2020
Last Updated
October 23, 2023
Sponsor
Stanford University
Collaborators
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
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1. Study Identification

Unique Protocol Identification Number
NCT04669158
Brief Title
Study of Oral Idebenone to Treat Non-Alcoholic Steatohepatitis
Official Title
A Pilot Study to Treat Adults With Non-Alcoholic Steatohepatitis With Oral Idebenone
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
July 30, 2021 (Actual)
Primary Completion Date
September 2024 (Anticipated)
Study Completion Date
September 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Stanford University
Collaborators
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Non Alcoholic Steatohepatitis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
45 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Idebenone
Arm Type
Experimental
Arm Description
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.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Idebenone
Other Intervention Name(s)
Placebo
Intervention Description
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.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
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.
Primary Outcome Measure Information:
Title
Number of Participants with Treatment-Related Adverse Events as Assessed by CTCAE v4.0
Description
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.
Time Frame
Week 60: Assessment of AEs
Secondary Outcome Measure Information:
Title
Change in fibrosis stage
Description
Number of Participants with change in fibrosis stage
Time Frame
Week 48: fibrosis change

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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<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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Natalie Torok, MD
Organizational Affiliation
Stanford University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Digestive Health Center, Stanford University
City
Redwood City
State/Province
California
ZIP/Postal Code
94063
Country
United States
Facility Name
Stanford University
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
Undecided

Learn more about this trial

Study of Oral Idebenone to Treat Non-Alcoholic Steatohepatitis

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