A Study of Experimental Obeticholic Acid For Primary Biliary Cholangitis (inflammation and scarring of liver bile ducts)

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Age: 18 years or older
Gender:
Any
Healthy Volunteers: No
Keywords: Primary Biliary Cholangitis, Moderate to Severe Hepatic Impairment, liver function, liver
Type: Drug study, Phase 4
Target:
4 Participants
Investigator:
Description
This study is for people with primary biliary cholangitis (also known as primary biliary cirrhosis or PBC). Because of your condition, you may be eligible for a research study on an investigational drug for your condition.

The Sponsor of this study, Intercept Pharmaceuticals, Inc., is trying to learn more about obeticholic acid (also known as OCA). We want to know its effect on your PBC condition when given with ursodeoxycholic acid (UDCA).

OCA (Ocaliva) has been approved in several countries (including the United States, Canada, and Europe) for the treatment of PBC. As an approved drug, OCA may be commercially available, or can be purchased to treat patients with your condition, PBC.

In this study, OCA is an investigational drug (also called a study drug). This means that OCA is being tested to obtain more information about its effects on PBC. The investigational drug, OCA, is a modified (manmade) version of a natural compound made in the liver called a bile acid. Bile acids are used by the body to help with digestion and have additional effects on liver function.

The purpose of this study is to find out how study drug, OCA, is absorbed and distributed through the body. We hope to determine the chemical changes of the study drug while it is in the body, and identify how the study drug is excreted out of the body. This is called pharmacokinetics, or PK. The Sponsor will also be studying the safety of OCA.
This study requires

The study team will make sure you qualify to take part in the study. Qualified participants will receive detailed information about the study. This may include a list of study-related tests and procedures.

Who can participate

Inclusion criteria:

  1. Definite or probable PBC diagnosis (consistent with American Association for the Study of Liver Diseases [AASLD] and the European Association for the Study of the Liver [EASL] practice guidelines; Lindor 2009; EASL 2009), as demonstrated by the presence of ≥2 of the following 3 diagnostic factors:
  2. History of elevated Alkaline phosphatase levels for at least 6 months prior to Day 0
  3. Positive antimitochondrial antibody (AMA) titer or if AMA negative or in low titer (<1:80) PBC-specific antibodies (anti-GP210 and/or anti-SP100 and/or antibodies against the major M2 components [PDC-E2, 2-oxo-glutaric acid dehydrogenase complex])
  4. Liver biopsy consistent with PBC
  5. A mean total bilirubin >ULN and ≤5x ULN and/or a mean ALP >3x ULN
  6. Age ≥18 years
  7. Either is not taking UDCA (no UDCA dose in the past ≥3 months) or has been taking UDCA for at least 12 months with a stable dose for ≥3 months prior to Day 0
  8. Contraception: Female subjects of childbearing potential must use ≥1 effective method of contraception during the trial and until 30 days following the last dose of investigational product. Effective methods of contraception are considered to be:
  9. Hormonal (e.g. contraceptive pill, patch, intramuscular implant or injection; or
  10. Double barrier method, i.e. (a) condom (male or female) or (b) diaphragm, with spermicide; or
  11. Intrauterine device (IUD); or
  12. Vasectomy (partner); or
  13. Abstinence, if in line with the preferred and usual lifestyle of the subject
  14. Must provide written informed consent and agree to comply with the study protocol

Exclusion criteria:

  1. History or presence of other concomitant liver diseases including:
  2. Hepatitis C virus infection
  3. Active Hepatitis B infection; however, subjects who have seroconverted (hepatitis B surface antigen and hepatitis B e antigen negative) may be included in this study after consultation with the medical monitor
  4. Primary sclerosing cholangitis (PSC)
  5. Alcoholic liver disease
  6. Definite autoimmune liver disease or overlap hepatitis
  7. Nonalcoholic steatohepatitis (NASH)
  8. Gilbert's Syndrome
  9. Presence of clinical complications of PBC or clinically significant hepatic decompensation, including:
  10. History of liver transplant, current placement on a liver transplant list, or current Model of End Stage Liver Disease (MELD) score >12. Subjects who are placed on a transplant list despite a relatively early disease stage (for example per regional guidelines) may be eligible as long as they do not meet any of the other exclusion criteria
  11. Cirrhosis with complications, including history (within the past 12 months) or presence of:
  12. Variceal bleed
  13. Uncontrolled ascites
  14. Encephalopathy
  15. Spontaneous bacterial peritonitis
  16. Known or suspected HCC
  17. Prior transjugular intrahepatic portosystemic shunt procedure
  18. Hepatorenal syndrome (type I or II) or screening (Visit 1 or 2) serum creatinine

    2 mg/dL (178 μmol/L)

  19. Mean total bilirubin >5x ULN
  20. Subjects who have undergone gastric bypass procedures (gastric lap band is acceptable) or ileal resection or plan to undergo either of these procedures
  21. Other medical conditions that may diminish life expectancy, including known cancers (except carcinomas in situ or other stable, relatively benign conditions such as chronic lymphocytic leukemia)
  22. If female: plans to become pregnant, known pregnancy or a positive urine pregnancy test (confirmed by a positive serum pregnancy test), or lactating
  23. Known history of human immunodeficiency virus infection
  24. Medical conditions that may cause nonhepatic increases in ALP (eg, Paget's disease or fractures within 3 months prior to Day 0)
  25. Other clinically significant medical conditions that are not well controlled or for which medication needs are anticipated to change during the study
  26. History of alcohol abuse or other substance abuse within 1 year prior to Day 0
  27. Participation in another investigational product, biologic, or medical device study within 30 days prior to Screening. Participation in a previous study of OCA is allowed with 3 months washout prior to enrollment in this study
  28. Mental instability or incompetence, such that the validity of informed consent or ability to be compliant with the study is uncertain
  29. History of known or suspected clinically significant hypersensitivity to OCA or any of its components
  30. UDCA naïve (unless contraindicated)
Benefits and risks of participating
BENEFITS:

We cannot promise any benefits to you as a result of taking part in this research study.


RISKS:

The study doctor will discuss the risks associated with taking part in this research study.
Compensation
If you agree to take part in this research study, we will compensate you $65.00 for your time and effort. Subjects will receive their compensation quarterly in the form of a check by mail. If you discontinue early from the study, you will receive partial payment based on the study visits you completed.
Resources
Schedule
Study duration and period
We expect that you will be in this research study for approximately 3 years.
Recruitment period
From Aug. 9, 2018
Location
UC Davis Medical Center
2000 Stockton Boulevard
Suite 100
Sacramento, CA 95817
Contact
Sandeep Dhaliwal
Research Topic
Conditions:
  • Liver Cirrhosis
  • Biliary