Trial Outcomes & Findings for Study of Oral Idebenone to Treat Non-Alcoholic Steatohepatitis (NCT NCT04669158)
NCT ID: NCT04669158
Last Updated: 2026-04-24
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
53 participants
Primary outcome timeframe
60 weeks
Results posted on
2026-04-24
Participant Flow
53 participants signed informed consent; 44 were allocated to treatment.
Participant milestones
| Measure |
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.
|
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.
|
|---|---|---|
|
Overall Study
NOT COMPLETED
|
5
|
3
|
|
Overall Study
STARTED
|
29
|
15
|
|
Overall Study
COMPLETED
|
24
|
12
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study of Oral Idebenone to Treat Non-Alcoholic Steatohepatitis
Baseline characteristics by cohort
| Measure |
Idebenone
n=24 Participants
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.
|
Placebo
n=12 Participants
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.
|
Total
n=36 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
56.5 years
STANDARD_DEVIATION 11.5 • n=2 Participants
|
60.8 years
STANDARD_DEVIATION 17.6 • n=1 Participants
|
57.8 years
STANDARD_DEVIATION 1.6 • n=3 Participants
|
|
Age, Customized
≤ 60 years
|
10 Participants
n=2 Participants
|
5 Participants
n=1 Participants
|
15 Participants
n=3 Participants
|
|
Age, Customized
> 60 years
|
14 Participants
n=2 Participants
|
7 Participants
n=1 Participants
|
21 Participants
n=3 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=2 Participants
|
4 Participants
n=1 Participants
|
17 Participants
n=3 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=2 Participants
|
8 Participants
n=1 Participants
|
19 Participants
n=3 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=2 Participants
|
1 Participants
n=1 Participants
|
4 Participants
n=3 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
18 Participants
n=2 Participants
|
11 Participants
n=1 Participants
|
29 Participants
n=3 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=2 Participants
|
0 Participants
n=1 Participants
|
3 Participants
n=3 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=2 Participants
|
0 Participants
n=1 Participants
|
0 Participants
n=3 Participants
|
|
Race (NIH/OMB)
Asian
|
7 Participants
n=2 Participants
|
4 Participants
n=1 Participants
|
11 Participants
n=3 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=2 Participants
|
0 Participants
n=1 Participants
|
0 Participants
n=3 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=2 Participants
|
0 Participants
n=1 Participants
|
0 Participants
n=3 Participants
|
|
Race (NIH/OMB)
White
|
10 Participants
n=2 Participants
|
8 Participants
n=1 Participants
|
18 Participants
n=3 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=2 Participants
|
0 Participants
n=1 Participants
|
0 Participants
n=3 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
7 Participants
n=2 Participants
|
0 Participants
n=1 Participants
|
7 Participants
n=3 Participants
|
|
Region of Enrollment
United States
|
24 Participants
n=2 Participants
|
12 Participants
n=1 Participants
|
36 Participants
n=3 Participants
|
PRIMARY outcome
Timeframe: 60 weeksPopulation: Participants who completed the study
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.
Outcome measures
| Measure |
Idebenone
n=24 Participants
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.
|
Placebo
n=12 Participants
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.
|
|---|---|---|
|
Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0
|
9 Participants
|
4 Participants
|
SECONDARY outcome
Timeframe: Week 48Population: Participants with week 48 data
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.
Outcome measures
| Measure |
Idebenone
n=21 Participants
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.
|
Placebo
n=12 Participants
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.
|
|---|---|---|
|
Change in Magnetic Resonance Elastography (MRE) as a Measure of Change in Fibrosis Stage
|
-1.47 kPa
Standard Deviation 0.078
|
0.833 kPa
Standard Deviation 0.09
|
Adverse Events
Idebenone
Serious events: 1 serious events
Other events: 9 other events
Deaths: 1 deaths
Placebo
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Idebenone
n=29 participants at risk
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.
|
Placebo
n=15 participants at risk
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.
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Choking
|
3.4%
1/29 • 60 weeks
|
0.00%
0/15 • 60 weeks
|
Other adverse events
| Measure |
Idebenone
n=29 participants at risk
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.
|
Placebo
n=15 participants at risk
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.
|
|---|---|---|
|
Gastrointestinal disorders
Diarrhea
|
13.8%
4/29 • 60 weeks
|
6.7%
1/15 • 60 weeks
|
|
Gastrointestinal disorders
Nausea
|
10.3%
3/29 • 60 weeks
|
6.7%
1/15 • 60 weeks
|
|
Gastrointestinal disorders
Bloating
|
10.3%
3/29 • 60 weeks
|
0.00%
0/15 • 60 weeks
|
|
Gastrointestinal disorders
Vomiting
|
3.4%
1/29 • 60 weeks
|
6.7%
1/15 • 60 weeks
|
|
Eye disorders
Ocular discomfort
|
6.9%
2/29 • 60 weeks
|
0.00%
0/15 • 60 weeks
|
|
Gastrointestinal disorders
Constipation
|
6.9%
2/29 • 60 weeks
|
0.00%
0/15 • 60 weeks
|
|
Gastrointestinal disorders
Abdominal pain
|
3.4%
1/29 • 60 weeks
|
6.7%
1/15 • 60 weeks
|
|
Gastrointestinal disorders
Hematochezia
|
3.4%
1/29 • 60 weeks
|
0.00%
0/15 • 60 weeks
|
|
Injury, poisoning and procedural complications
Injured wrist
|
3.4%
1/29 • 60 weeks
|
0.00%
0/15 • 60 weeks
|
|
General disorders
Fatigue
|
0.00%
0/29 • 60 weeks
|
6.7%
1/15 • 60 weeks
|
|
Ear and labyrinth disorders
Sore throat
|
3.4%
1/29 • 60 weeks
|
0.00%
0/15 • 60 weeks
|
|
Nervous system disorders
Headache
|
3.4%
1/29 • 60 weeks
|
0.00%
0/15 • 60 weeks
|
|
Infections and infestations
COVID-19 Infection
|
3.4%
1/29 • 60 weeks
|
0.00%
0/15 • 60 weeks
|
|
Musculoskeletal and connective tissue disorders
Flank pain
|
3.4%
1/29 • 60 weeks
|
0.00%
0/15 • 60 weeks
|
|
General disorders
Fever
|
3.4%
1/29 • 60 weeks
|
0.00%
0/15 • 60 weeks
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
3.4%
1/29 • 60 weeks
|
0.00%
0/15 • 60 weeks
|
|
Renal and urinary disorders
Urine color changes
|
3.4%
1/29 • 60 weeks
|
0.00%
0/15 • 60 weeks
|
|
Blood and lymphatic system disorders
Edema
|
3.4%
1/29 • 60 weeks
|
0.00%
0/15 • 60 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Nasal Congestion
|
0.00%
0/29 • 60 weeks
|
6.7%
1/15 • 60 weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place