Study of Obeticholic Acid (OCA) Evaluating Pharmacokinetics and Safety in Participants With Primary Biliary Cholangitis (PBC) and Hepatic Impairment

Stopped early · Phase 4 · Has a placebo group

Conditions studied: Liver Cirrhosis, Biliary

In brief

This Phase 4, randomized, double-blind, placebo-controlled study will evaluate the pharmacokinetics (PK) and safety of OCA treatment in participants with PBC and moderate to severe hepatic impairment over a 48-week treatment period. Participants who have completed their 48-week double blind treatment period will continue double-blind treatment until all randomized participants have completed their 48-week treatment period and the database for that period is locked. An open-label extension study in which all participants receive OCA will be considered following review of blinded safety and PK data.

Key facts

Study ID

NCT03633227

Run by

Intercept Pharmaceuticals

People needed

22

Starts

2018-06-22

Expected to finish

2021-07-09

Last updated by the study team

2022-09-06

Who can join

Age: 18 and older, up to 85. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• A definite or probable diagnosis of PBC (consistent with American Association for the Study of Liver Diseases [AASLD] and European Association for the Study of the Liver [EASL] Practice Guidelines, defined as having ≥2 of the following 3 diagnostic factors:
• History of elevated alkaline phosphatase (ALP) levels for at least 6 months
• Positive antimitochondrial antibody (AMA) titer or if AMA negative or low titer (≤1:80), PBC specific antibodies (anti-glycoprotein 210 [GP210] and/or anti-SP100) and/or antibodies against the major M2 components (E2 component of mitochondrial pyruvate dehydrogenase complex [PDC-E2], 2-oxo-glutaric acid dehydrogenase complex)
• Liver biopsy consistent with PBC (collected at any time prior to Screening)
• Evidence of cirrhosis including at least one of the following:
• Biopsy results consistent with PBC Stage 4
• Liver stiffness as assessed by Transient Elastography (TE) Median Value ≥16.9 kilopascals (kPa)
• Clinical evidence in the absence of acute liver failure consistent with cirrhosis including: gastroesophageal varices, ascites, radiological evidence of cirrhosis (nodular liver or enlargement of portal vein and splenomegaly)
• Combined low platelet count (<140,000/cubic millimeter [mm\^3]) with
• persistent decrease in serum albumin, or
• elevation in prothrombin time/international normalized ratio (INR) (not due to antithrombotic agent use), or
• elevated bilirubin (2*upper limit of normal [ULN])
• Satisfy the criteria of the modified Child-Pugh (CP) classification for hepatic impairment during Screening:
• Moderate: CP-B (Scores 7 to 9) or
• Severe: CP-C (Scores 10 to 12)
• Model of end-stage liver disease (MELD) score of 6 to 24 at Screening
• Taking ursodeoxycholic acid (UDCA) for at least 12 months (stable dose for ≥3 months) prior to Day 1, or unable to tolerate or unresponsive to UDCA (no UDCA for ≥3 months)

You may not qualify if…

• Non-cirrhotic or cirrhotic CP-A (Mild; Score 5 to 6)
• History of liver transplant or organ transplant
• History of alcohol or drug abuse within 12 months prior to Screening
• Hepatic encephalopathy (as defined by a West Haven score of ≥2
• History or presence of other concomitant liver diseases including:
• Hepatitis C virus infection and ribonucleic acid (RNA) positive
• Active hepatitis B infection; however, participants 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
• Alcoholic liver disease
• Definite autoimmune liver disease or overlap hepatitis
• Gilbert's Syndrome
• In the opinion of the Investigator, fluctuating or rapidly deteriorating hepatic function prior to randomization
• Other inclusion/exclusion criteria may apply.

Where it is running

• Inland Empire Liver Foundation — Rialto, California, United States
• University of California, Ddavis Medical Center — Sacramento, California, United States
• Schiff Center for Liver Diseases/ University of Miami — Miami, Florida, United States
• Mercy Medical Center — Baltimore, Maryland, United States
• University Of Michigan — Ann Arbor, Michigan, United States
• Kansas City Research Institute — Kansas City, Missouri, United States
• The Ohio State University Wexner Medical Center — Columbus, Ohio, United States
• UPMC Center for Liver Diseases — Pittsburgh, Pennsylvania, United States
• The Liver Institute at Methodist Dallas Medical Center — Dallas, Texas, United States
• Baylor College of Medicine- Advanced Liver Therapies — Houston, Texas, United States
• American Research Corporation at theTexas Liver Institute — San Antonio, Texas, United States
• Hospital Italiano de Buenos Aires — Buenos Aires, Argentina
• Hospital Universitario Austral — Buenos Aires, Argentina
• Hospital Aleman — Caba, Argentina
• Hospital Britanico de Buenos Aires — Caba, Argentina
• Hospital de Gastroenterologia "Dr Carlos Bonorino Udaondo" — Ciudad Autonoma de Buenos Aire, Argentina
• Higea S.A. — Mendoza, Argentina
• Hospital Universitario Austral — Pilar, Argentina
• Hospital Provincial del Centenario — Rosario, Argentina
• Royal Prince Alfred Hospital — Camperdown, Australia
• Nepean Hospital — Kingswood, Australia
• CUB Hospital Erasme — Brussels, Belgium
• Universitair Ziekenhuis Antwerpen UZA — Edegem, Belgium
• University Hospital Leuven — Leuven, Belgium
• Hospital das Clínicas da Universidade Federal de Minas Gerais - UFMG — Belo Horizonte, Brazil

Full record on ClinicalTrials.gov

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