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A Study to Compare MitoQ and Placebo to Treat Non-alcoholic Fatty Liver Disease (NAFLD)

Phase 2
Terminated
Conditions
Non-alcoholic Fatty Liver Disease
Interventions
Registration Number
NCT01167088
Lead Sponsor
Antipodean Pharmaceuticals, Inc.
Brief Summary

The purpose of this study is to investigate whether a new medicine, called mitoquinone, will reduce raised liver enzymes due to NAFLD and to see if it is safe.

Detailed Description

Not available

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
110
Inclusion Criteria
  1. Provide written informed consent
  2. NAFLD as determined by raised ALT (> 1.5 x ULN corresponding to >29U/L for females and >45U/L for males] in the screening period and on at least two other occasions in the previous 6 months) and ultrasound evidence of steatosis (in the previous 12 months).
  3. Be aged between 18 - 70 years on the day of consent
  4. Expect to not require or make any changes in all their current concomitant medications (prescribed and over-the-counter) for the duration of their participation in the study
  5. Female patients with reproductive potential must have a negative serum pregnancy test within 14 days prior to start of trial and must agree to use a medically acceptable method of contraception throughout the treatment period and for 1 month after discontinuation of treatment. Acceptable methods of contraception include IUD, oral contraceptive, subdermal implant and double barrier (condom with a contraceptive sponge or contraceptive pessary)
Exclusion Criteria
  1. Alcohol consumption > 14 units/week for females and 21 units/week for males
  2. Hepatocellular carcinoma (HCC) or suspicion of HCC
  3. Presence of human immunodeficiency virus (HIV), Hepatitis B (HBV) or Hepatitis C (HCV)
  4. Renal impairment (creatinine > 1.5 x ULN) or hepatorenal syndrome
  5. Chronic pancreatitis
  6. Hospitalization for liver disease within 60 days of the baseline visit
  7. Previously diagnosed diabetes / treatment with insulin sensitizing agents
  8. Severe or morbid obesity (BMI>40kg/m2)
  9. ALT or AST > 10 times ULN
  10. Liver transplant recipients
  11. Corticosteroids in the past 30 days
  12. Any participant who has received any investigational drug or device within 30 days of dosing, or who is scheduled to receive another investigational drug or device during the course of this trial
  13. A history of a malignancy other than treated basal cell or squamous cell carcinoma of the skin; those with a history of malignancy that has been treated with no recurrence within the last 2 years are not excluded
  14. Females who are pregnant or breastfeeding
  15. Use of Coenzyme Q10, either prescribed or purchased over-the-counter, are prohibited during the study, except for doses of up to 25mg/day which have been stable for 30 days prior to baseline. Higher doses require a 7 day washout prior to baseline.
  16. Use of Vitamin E, either prescribed or purchased over-the-counter, are prohibited during the study, except for doses of up to 200IU/day which have been stable for 30 days prior to baseline. Higher doses require a 90 day washout prior to baseline.
  17. Any changes to prescription medication in the 30 days prior to baseline
  18. A history of a hypersensitivity reaction to any components of the study drug or structurally similar compounds including Coenzyme Q10 and idebenone
  19. Unable to swallow tablets whole
  20. Patients with histological or clinical evidence of established cirrhosis
  21. Suffering from any other disease or condition which, in the opinion of the investigator, means that it would not be in the patient's best interest to participate in this study

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Mitoquinone mesylate tablets (MitoQ)Mitoquinone mesylate-
Matching placebo tabletPlacebo-
Primary Outcome Measures
NameTimeMethod
Percentage change in ALT (relative to baseline) at the end of the treatment period (Day 90) for MitoQ compared with placebo.Baseline and 3 months
Secondary Outcome Measures
NameTimeMethod
Absolute change in ALT level (relative to baseline) at end of treatment period for MitoQ compared with placeboBaseline and 3 months
The percentage of participants whose ALT levels are in the normal range at the end of the treatment period.3 months
The difference in the percentage and absolute rates of change in ALT levels between MitoQ and placebo.Baseline to 3 months
The percentage and absolute change in AST at the end of the treatment period (relative to baseline) for MitoQ compared with placebo.Baseline and 3 months
The change in HOMA-IR at the end of the treatment period (relative to baseline) for MitoQ compared with placebo.Baseline and 3 months
The change in HbA1c at the end of the treatment period (relative to baseline) for MitoQ compared with placebo.Baseline and 3 months
The percentage and absolute change in GGT and alkaline phosphatase at the end of the treatment period (relative to baseline) for MitoQ compared with placebo.Baseline and 3 months
Areas under the ALT, AST, GGT and alkaline phosphatase curves from baseline to the end of the treatment period.Baseline to 3 months
The change in markers of liver inflammation (leptin and adiponectin) at the end of the treatment period (relative to baseline) for MitoQ compared with placebo.Baseline and 3 months
The change in biomarkers of mitochondrial function and oxidative damage (isoprostanes) at the end of the treatment period (relative to baseline) for MitoQ compared with placebo.Baseline and 3 months
The change in blood pressure at the end of the treatment period (relative to baseline) for MitoQ compared with placebo.Baseline and 3 months
The change in blood lipid profile at the end of the treatment period (relative to baseline) for MitoQ compared with placebo.Baseline and 3 months
Incidence of adverse eventsBaseline to Follow-up (total 4 months)
Clinically relevant deterioration in laboratory variablesBaseline to Follow-up (total 4 months)
Clinically relevant deterioration in vital signsBaseline to Follow-up (total 4 months)
Clinically relevant deterioration in ECG parametersBaseline to Follow-up (total 4 months)

Trial Locations

Locations (1)

Freeman Hospital

🇬🇧

Newcastle-upon-Tyne, United Kingdom

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