Primary Biliary Cholangitis (PBC) Clinical Trials

Primary Biliary Cholangitis is a chronic progressive autoimmune liver disease characterized by destruction of intrahepatic bile ducts. The pathophysiology involves loss of tolerance to self-antigens on mitochondrial proteins (particularly PDC-E2, a component of the pyruvate dehydrogenase complex), leading to autoimmune attack primarily on small to medium-sized bile ducts. This triggers both innate and adaptive immune responses including autoreactive T cells and B cells producing antimitochondrial antibodies (AMA).

The most common symptoms of Primary Biliary Cholangitis include Fatigue, often severe and disproportionate to liver function, Pruritus causing intense skin itching, Jaundice with yellowing of skin and eyes, Abdominal pain and bloating, Osteoporosis and bone pain, among others. About 14.6 per 100,000 people worldwide in a 2021 pooled analysis of 47 population studies, highest in North America (21.8 per 100,000) and lowest in the Asia-Pacific region (9.8 per 100,000). Rates are rising in all regions (Lv et al., J Gastroenterol Hepatol 2021). Symptoms can range from mild to severe and may vary between individuals.

Trial Friend lists all active clinical trials for Primary Biliary Cholangitis from ClinicalTrials.gov, with eligibility criteria translated into plain English. Patients can filter by trial phase and recruitment status, search by location, and use the Match Me tool to score trials against their profile. Research in Primary Biliary Cholangitis is advancing treatment options for patients who don't respond well to traditional ursodeoxycholic acid therapy. Saroglitazar, a PPAR alpha and gamma agonist from Zydus, faces an FDA decision on November 27, 2026.

Yes. As of October 21, 2026, 4 FDA-approved medicines have a current label that names Primary Biliary Cholangitis: Iqirvo (elafibranor), first approved in 2024; Livdelzi (seladelpar), first approved in 2024; Lynavoy (linerixibat), first approved in 2026; and Ursodiol tablets (ursodiol). Corticosteroids and immune globulins count once each. The FDA is due to decide on Saroglitazar by November 27, 2026; the FDA calendar page carries the outcome the day it lands.

The exact cause of Primary Biliary Cholangitis is not fully understood. It is not caused by a single gene; inherited variants raise risk, and immune, environmental or other triggers are thought to set it off. Autoimmune with familial clustering but no diagnostic gene; anti-mitochondrial antibody is the key test. About 14.6 per 100,000 people worldwide in a 2021 pooled analysis of 47 population studies, highest in North America (21.8 per 100,000) and lowest in the Asia-Pacific region (9.8 per 100,000). Rates are rising in all regions (Lv et al., J Gastroenterol Hepatol 2021). Research is ongoing to better understand the disease mechanism.

No single gene test diagnoses Primary Biliary Cholangitis. The diagnosis rests on clinical findings and laboratory or imaging results; genetic variants shift risk rather than cause the disease. Autoimmune with familial clustering but no diagnostic gene; anti-mitochondrial antibody is the key test.

Some trials collect genetic samples for research or check specific risk markers. The Genetic Testing section of this page shows any tests listed in the NIH Genetic Testing Registry, which for this condition are usually research or risk-marker tests rather than diagnostic ones.

Eligibility for Primary Biliary Cholangitis clinical trials depends on factors like age, diagnosis, treatment history, and overall health. Each trial sets its own inclusion and exclusion criteria. Trial Friend translates these requirements from medical language into plain English so patients can review them more easily. The Match Me tool can score your profile against active trials to help identify studies that may be a fit.

Participating in a Primary Biliary Cholangitis clinical trial typically involves scheduled visits, medical tests, and close monitoring by a research team. Depending on the study phase, you may receive an investigational treatment, a placebo, or the current standard of care. Trials may last from a few weeks to several years. Study participation is voluntary, and you can withdraw at any time.