Primary Biliary Cholangitis (PBC) Study

STUDY BASICS

Have you been diagnosed with PBC (formerly called primary biliary cirrhosis) and are you aged 18-75? If so, you may be eligible to participate in a research study to help find out if an investigational study drug is a safe and effective treatment for people with PBC.

STUDY PURPOSE

PBC is an uncommon autoimmune disease caused by damage to the bile ducts in the liver. Although many people with PBC do not initially have symptoms, ongoing liver damage often causes excessive scarring called fibrosis. Over time, as scar tissue replaces healthy liver tissue, fibrosis can progress to cirrhosis—a serious condition that increases the risk of liver failure and liver cancer. Treatments for PBC exist, but they do not work for everyone. The purpose of this study is to find out if an investigational study drug is safe and effective for treating people with PBC and to help researchers determine which dose of the study drug works best. The study drug is considered to be investigational because it has not been approved by the United States Food and Drug Administration. Researchers hope their findings will lead to better treatments for people with PBC in the future.

COULD THIS STUDY BE RIGHT FOR YOU?

• Ages 18 to 75
• Diagnosed with primary biliary cholangitis (sometimes called primary biliary cirrhosis)
• Not diagnosed with hepatitis B, hepatitis C, or HIV
• Taking a stable dose of UDCA (12-20 mg/kg/day) for at least 6 months, or are not able to take UDCA
• Not pregnant

WHAT PARTICIPANTS CAN EXPECT

The Intrepid study will consist of a Screening Period (up to 28 days), a Study Treatment Period (12 weeks) and a Safety Follow-up Period (28 days). The study requires approximately 8 visits to the clinic over a period of approximately 20 weeks. The majority of study participants will receive one of two oral doses of the investigational drug; 1 out of 7 subjects will receive placebo (a tablet that looks like the investigational drug, but does not have any active ingredients). Both the study drug and the placebo are pills taken daily by mouth. Tests and procedures during the study include blood draws, physical exams, fibroscan, urine sample, electrocardiogram, quality of life questionnaires, and others as needed. Reimbursement may be provided for study-related travel and expenses.