Emricasan, a Caspase Inhibitor, for Treatment of Subjects With Decompensated NASH Cirrhosis

Overview

  • Study type

    Interventional
  • Study phase

    II
  • Study IDs

  • Describes the nature of a clinical study. Types include:

    • Observational study — observes people and measures outcomes without affecting results.
    • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
    • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
  • During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

  • Site IRB
    • Rochester, Minnesota: 17-001485
    NCT ID: NCT03205345
    Sponsor Protocol Number: IDN-6556-17

About this study

This is a multicenter, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of emricasan in improving event-free survival based on a composite clinical endpoint (where all-cause mortality, new decompensation events, and MELD score progression are events) in subjects with decompensated NASH cirrhosis.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. If you need assistance understanding the eligibility criteria, please contact the study team.

See eligibility criteria

Inclusion Criteria:

  1. Male or female subjects 18 years or older, able to provide written informed consent and able to understand and willing to comply with the requirements of the study.
  2. Cirrhosis due to NASH with exclusion of other causes of cirrhosis (e.g. chronic viral hepatitis, alcoholic liver disease, etc.)
  3. At least one of the following: a) history of variceal hemorrhage (more than 3 months prior to day 1) documented on endoscopy and requiring blood transfusion, b) history of at least moderate ascites (on physical exam or imaging) currently treated with diuretics.
  4. MELD score ≥12 and ≤20 during screening
  5. Albumin ≥2.5 g/dL during screening
  6. Serum creatinine ≤1.5 mg/dL during screening

Exclusion Criteria:

  1. Evidence of severe decompensation
  2. Non-cirrhotic portal hypertension
  3. Child-Pugh score ≥10
  4. Current use of anticoagulants that affect prothrombin time or international normalized ratio
  5. ALT >3 times upper limit of normal (ULN) or AST >5 times ULN during screening
  6. Initiation or discontinuation of non-selective beta blockers within 1 month of screening
  7. Transjugular intrahepatic portosystemic shunt or other porto-systemic bypass procedure within 1 year of screening or previously requiring revision
  8. Alpha-fetoprotein >50 ng/mL in the last year
  9. History of hepatocellular carcinoma (HCC) or evidence of HCC
  10. History of malignancies other than HCC, unless successfully treated with curative intent and believed to be cured
  11. Prior liver transplant
  12. Uncontrolled diabetes mellitus (HbA1c >9%)
  13. Change in diabetes medications or vitamin E within 3 months of screening
  14. Restrictive bariatric surgery or bariatric device within 1 year of screening or prior malabsorptive bariatric surgery
  15. Symptoms of biliary colic unless resolved following cholecystectomy
  16. History of significant alcohol consumption within the past 5 years
  17. Current use of medications that are considered inhibitors of organic anion transporting polypeptide OATP1B1 and OATP1B3 transporters
  18. Prolongation of screening (pre-treatment) QTcF interval of >500 msecs, or history or presence of clinically concerning cardiac arrhythmias
  19. Significant systemic or major illness other than liver disease
  20. Human immunodeficiency virus infection
  21. Use of alcohol, controlled substances (including inhaled or injected drugs), or non-prescribed use of prescription drugs within 1 year of screening to the point of interfering with the subject's ability to comply with study procedures and study drug administration in the investigator's judgement

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Douglas Simonetto, M.D.

Contact us for the latest status

Contact information:

Amy Olofson R.N.

(507)284-2638

Olofson.Amy@mayo.edu