Last updated on July 2019

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


Brief description of study

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 requiring liver transplantation or resulting in death. The investigational drug, Obeticholic Acid (OCA) is a modified bile acid and FXR agonist that is derived from the primary human bile acid chenodeoxycholic acid. The key mechanisms of action of OCA, including its choleretic, anti-inflammatory, and anti-fibrotic properties, underlie its hepatoprotective effects and result in attenuation of injury and improved liver function in a cholestatic liver disease such as PBC. The study will assess the effect of OCA compared to placebo, combined with stable standard care, on clinical outcomes in PBC patients.

Detailed Study 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 subjects with PBC. The study will include a screening period of up to 8 weeks, requiring two clinic visits. Eligible participants will be randomly allocated (1:1) to treatment with either OCA 5 mg or matching placebo tablets, taken orally once daily for the majority of subjects; dose and frequency will be modified for subjects with cirrhosis and classified as Child-Pugh B or C. Randomization will be stratified by standard treatment with UDCA (yes/no) and baseline liver function. The treatment period involves clinic visits approximately every 3 months. At the 3 month visit or any study visit thereafter, if study treatment is tolerated, participants' dose should be titrated per protocol in a blinded manner eg for participants who are non-cirrhotic or classified as Child-Pugh A and randomized to OCA, they should receive the maximum daily dose of 10 mg OCA, those on placebo continue to receive placebo. Subsequently, daily dosage may return to 5 mg if necessary for these participants who are non-cirrhotic or classified as Child-Pugh A, but should be increased to 10 mg if possible, based on tolerability and clinical judgment. Safety and tolerability will be assessed by monitoring adverse events and vital signs, and blood and urine testing. The study is event driven and total duration will be determined by the time required to accrue approximately 127 primary endpoint events, estimated to be approximately 10 years. Subjects are expected to have a minimum follow-up time of approximately 6 years.

Clinical Study Identifier: NCT02308111

Find a site near you

Start Over

Hospital Aleman

Buenos Aires, Argentina
  Connect »

Indiana University

Indianapolis, IN United States
  Connect »

UT Health

Houston, TX United States
  Connect »

Kansas City Research Institute

Kansas City, MO United States
  Connect »

University of Chicago

Chicago, IL United States
  Connect »

University of Miami

Miami, FL United States
  Connect »

Swedish Liver Clinic

Seattle, WA United States
  Connect »

Mercy Medical Center

Baltimore, MD United States
  Connect »

Tulane University Medical Center

New Orleans, LA United States
  Connect »

University of Louisville

Louisville, KY United States
  Connect »

Mayo Clinic

Phoenix, AZ United States
  Connect »

Hospital Italiano

Buenos Aires, Argentina
  Connect »

Dim Cl nica Privada

Ramos Mejía, Argentina
  Connect »

University of California Davis

Sacramento, CA United States
  Connect »

Cedars-Sinai Medical Center

Los Angeles, CA United States
  Connect »

McGuire DVAMC

Richmond, VA United States
  Connect »

Piedmont Atlanta Hospital

Atlanta, GA United States
  Connect »

Brooke Army Medical Center

Fort Sam Houston, TX United States
  Connect »

Quality Medical Research, PLLC

Nashville, TN United States
  Connect »

Mayo Clinic Arizona

Phoenix, AZ United States
  Connect »

Stanford University

Stanford, CA United States
  Connect »

UF Hepatology Research at CTRB

Gainesville, FL United States
  Connect »

Emory University Hospital

Atlanta, GA United States
  Connect »

UMass Medical School

Worcester, MA United States
  Connect »

Mount Sinai Beth Israel

New York, NY United States
  Connect »

Dim Cl nica Privada

Ramos Mejía, Argentina
  Connect »

Austin Hospital

Heidelberg, Australia
  Connect »

UZA

Edegem, Belgium
  Connect »

Uz Leuven

Leuven, Belgium
  Connect »

Chum

Montreal, QC Canada
  Connect »

Queen Mary Hospital

Hong Kong, Hong Kong
  Connect »

Rethy Pal Hospital

Bekescsaba, Hungary
  Connect »

Dim Cl nica Privada

Ramos Mejía, Argentina
  Connect »

Dim Cl nica Privada

Ramos Mejía, Argentina
  Connect »

Flinders Medical Centre

Bedford Park, Australia
  Connect »

Box Hill Hospital

Box Hill, Australia
  Connect »

Dim Cl nica Privada

Ramos Mejía, Argentina
  Connect »

Dim Cl nica Privada

Ramos Mejía, Argentina
  Connect »

Hospital Italiano de Buenos Aires

Ciudad Autónoma de Buenos Aires, Argentina
  Connect »

Dim Cl nica Privada

Ramos Mejía, Argentina
  Connect »

Hospital Italiano de Buenos Aires

Ciudad Autónoma de Buenos Aires, Argentina
  Connect »

Hospital Universitario Austral

Presidente Derqui, Argentina
  Connect »

Dim Cl nica Privada

Ramos Mejía, Argentina
  Connect »

Hospital Italiano de Buenos Aires

Ciudad Autónoma de Buenos Aires, Argentina
  Connect »

Hospital Aleman

Ciudad Autónoma de Buenos Aires, Argentina
  Connect »

Hospital Britanico De Buenos Aires

Ciudad Autónoma de Buenos Aires, Argentina
  Connect »

Cepec Huufma

Sao Luis, Brazil
  Connect »

Dim Cl nica Privada

Ramos Mejía, Argentina
  Connect »

Hospital Italiano de Buenos Aires

Ciudad Autónoma de Buenos Aires, Argentina
  Connect »

Hospital Aleman

Ciudad Autónoma de Buenos Aires, Argentina
  Connect »

Hospital Britanico De Buenos Aires

Ciudad Autónoma de Buenos Aires, Argentina
  Connect »