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| ID | Type | Description | Link |
|---|---|---|---|
| R21DK111217 | U.S. NIH Grant/Contract | View source |
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| Name | Class |
|---|---|
| National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | NIH |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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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):
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:
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
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| Name | Affiliation | Role |
|---|---|---|
| Natalie Torok, MD | Stanford University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Digestive Health Center, Stanford University | Redwood City | California | 94063 | United States | ||
| Stanford University |
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53 participants signed informed consent; 44 were allocated to treatment.
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| FG001 | 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. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Participant who completed the study
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| BG001 | Placebo |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Participants who completed the study | Posted | Count of Participants | Participants | 60 weeks |
|
60 weeks
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Choking | Respiratory, thoracic and mediastinal disorders | Systematic Assessment | Not related |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhea | Gastrointestinal disorders | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Natalie Torok, MD | Stanford University | (650) 498-7878 | ntorok@stanford.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Sep 15, 2021 | Nov 3, 2025 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D065626 | Non-alcoholic Fatty Liver Disease |
| D005355 | Fibrosis |
| ID | Term |
|---|---|
| D005234 | Fatty Liver |
| D008107 | Liver Diseases |
| D004066 | Digestive System Diseases |
| D010335 | Pathologic Processes |
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| ID | Term |
|---|---|
| C036619 | idebenone |
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| 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. |
|
| Week 48 |
| Stanford |
| California |
| 94305 |
| United States |
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. |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Age, Customized | Count of Participants | Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | 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.
|
|
| Secondary | 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. | Participants with week 48 data | Posted | Mean | Standard Deviation | kPa | Week 48 |
|
|
|
|
| 1 |
| 29 |
| 1 |
| 29 |
| 9 |
| 29 |
| EG001 | 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. | 0 | 15 | 0 | 15 | 4 | 15 |
|
| Nausea | Gastrointestinal disorders | Systematic Assessment |
|
| Bloating | Gastrointestinal disorders | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | Systematic Assessment |
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| Ocular discomfort | Eye disorders | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | Systematic Assessment |
|
| Hematochezia | Gastrointestinal disorders | Systematic Assessment |
|
| Injured wrist | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Fatigue | General disorders | Systematic Assessment |
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| Sore throat | Ear and labyrinth disorders | Systematic Assessment |
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| Headache | Nervous system disorders | Systematic Assessment |
|
| COVID-19 Infection | Infections and infestations | Systematic Assessment |
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| Flank pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Fever | General disorders | Systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Urine color changes | Renal and urinary disorders | Systematic Assessment |
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| Edema | Blood and lymphatic system disorders | Systematic Assessment |
|
| Nasal Congestion | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
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| D013568 |
| Pathological Conditions, Signs and Symptoms |