Status:

TERMINATED

Phase 4 Study of Obeticholic Acid Evaluating Clinical Outcomes in Patients With Primary Biliary Cholangitis

Lead Sponsor:

Intercept Pharmaceuticals

Conditions:

Liver Cirrhosis, Biliary

Eligibility:

All Genders

18+ years

Phase:

PHASE4

Brief Summary

Primary Biliary Cholangitis (PBC) is a serious, life-threatening, bile acid related liver disease of unknown cause. Without treatment, it frequently progresses to liver fibrosis and eventual cirrhosis...

Detailed Description

This Phase 4, double-blind, randomized, placebo-controlled, multicenter study is being undertaken at up to 170 sites internationally to evaluate the effect of OCA on clinical outcomes in 428 participa...

Eligibility Criteria

Inclusion

  • Key
  • 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:
  • History of elevated Alkaline phosphatase levels for at least 6 months
  • 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 \[PDC-E2, 2-oxo-glutaric acid dehydrogenase complex\])
  • Liver biopsy consistent with PBC
  • A mean total bilirubin \>ULN and ≤5x ULN and/or a mean ALP \>3x ULN
  • 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

Exclusion

  • History or presence of other concomitant liver diseases including:
  • Hepatitis C virus infection
  • 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
  • Primary sclerosing cholangitis (PSC)
  • Alcoholic liver disease
  • Definite autoimmune liver disease or overlap hepatitis
  • Nonalcoholic steatohepatitis (NASH)
  • Gilbert's Syndrome
  • Presence of clinical complications of PBC or clinically significant hepatic decompensation, including:
  • 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
  • Cirrhosis with complications, including history (within the past 12 months) or presence of:
  • Variceal bleed
  • Uncontrolled ascites
  • Encephalopathy
  • Spontaneous bacterial peritonitis
  • Known or suspected HCC
  • Prior transjugular intrahepatic portosystemic shunt procedure
  • Hepatorenal syndrome (type I or II) or screening (Visit 1 or 2) serum creatinine \>2 mg/dL (178 μmol/L)
  • Mean total bilirubin \>5x ULN
  • Subjects who have undergone gastric bypass procedures (gastric lap band is acceptable) or ileal resection or plan to undergo either of these procedures
  • Other medical conditions that may diminish life expectancy, including known cancers (except carcinomas in situ or other stable, relatively benign conditions)
  • If female: plans to become pregnant, known pregnancy or a positive urine pregnancy test (confirmed by a positive serum pregnancy test), or lactating
  • Known history of human immunodeficiency virus infection
  • Medical conditions that may cause nonhepatic increases in ALP (eg, Paget's disease or fractures within 3 months)
  • Other clinically significant medical conditions that are not well controlled or for which medication needs are anticipated to change during the study
  • History of alcohol abuse or other substance abuse within 1 year prior to Day 0
  • 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
  • Mental instability or incompetence, such that the validity of informed consent or ability to be compliant with the study is uncertain
  • History of known or suspected clinically significant hypersensitivity to OCA or any of its components
  • UDCA naïve (unless contraindicated)

Key Trial Info

Start Date :

December 26 2014

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

December 23 2021

Estimated Enrollment :

334 Patients enrolled

Trial Details

Trial ID

NCT02308111

Start Date

December 26 2014

End Date

December 23 2021

Last Update

March 9 2023

Active Locations (181)

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Page 1 of 46 (181 locations)

1

Mayo Clinic Arizona

Phoenix, Arizona, United States, 85054

2

Cedars-Sinai Medical Center

Los Angeles, California, United States, 90048

3

Inland Empire Liver Foundation

Rialto, California, United States, 92377

4

University of California Davis, Davis Medical Center

Sacramento, California, United States, 95817