New Treatments & Clinical Trials for Primary Biliary Cholangitis

Primary biliary cholangitis is a chronic liver disease where the immune system attacks bile ducts, causing bile to back up and damage liver tissue. About 65 out of 100,000 people in the U.S. have PBC, mostly women diagnosed in their 50s or 60s. Ursodiol has been the standard treatment for three decades, and obeticholic acid became the second approved drug in 2016.

What's actually going on in research

Trials are testing new drugs that target different bile acid pathways, anti-fibrotic agents to slow scarring, and combinations that might work better than single drugs. Researchers are also studying drugs that reduce itch, which affects most people with PBC and can be severe. Some trials are testing whether earlier treatment prevents progression to cirrhosis.

What to know before you search

Eligibility typically depends on alkaline phosphatase levels, whether you've tried ursodiol, liver biopsy or elastography results showing disease stage, and sometimes the presence and severity of itch.

What types of trials are currently open

• Treatment trials — Testing new drugs or combinations to improve liver enzyme levels and slow disease progression, typically in people who haven't responded adequately to ursodiol.
• Itch trials — Studies focused specifically on reducing pruritus, which can be debilitating and doesn't always improve with liver-directed treatment.
• Anti-fibrosis trials — Testing drugs that aim to reduce liver scarring directly, which could prevent progression to cirrhosis.
• Biomarker studies — Research to identify blood tests or imaging markers that predict disease progression and treatment response.
• Natural history studies — Long-term tracking of people with PBC to understand how the disease progresses and what factors affect outcomes.