Primary biliary cholangitis (PBC) is a chronic autoimmune liver disease in which the immune system slowly destroys the small bile ducts inside the liver, leading to bile buildup, inflammation, and over time cirrhosis. Most patients are women diagnosed in midlife, often discovered incidentally on routine blood tests. With early treatment, many people maintain near-normal liver function for decades.
What's actually going on in research
Ursodeoxycholic acid has been the mainstay of treatment for decades, slowing disease progression in most patients. Obeticholic acid is now approved for patients with inadequate response to ursodeoxycholic acid, and several newer agents — including fibrates, PPAR agonists, and FXR agonists — are in mid- to late-stage trials aiming to normalize liver enzymes and reduce the risk of liver transplant. Therapies targeting the itch (pruritus) caused by bile salt buildup are also advancing.
PPAR agonist therapy
Fibrates and next-generation PPAR agonists such as seladelpar are showing meaningful reductions in liver enzymes and itch in trials, offering a new option for patients who do not respond adequately to existing therapies.
FXR agonists
Obeticholic acid, an FXR agonist, is now approved for second-line use, and newer, better-tolerated FXR agonists are in trials aiming to improve response rates and reduce the pruritus side effect.
Pruritus management
Debilitating itch is a major quality-of-life burden in PBC, and trials are testing bile acid sequestrants, IBAT inhibitors, and other agents specifically targeting cholestatic pruritus.
What to know before you search
Eligibility typically requires confirmed PBC diagnosis, abnormal liver enzymes, and documented response or non-response to ursodeoxycholic acid.
What types of trials are currently open
- Second-line therapy trials — Testing PPAR agonists and next-generation FXR agonists in patients with inadequate ursodeoxycholic acid response.
- Combination therapy trials — Evaluating ursodeoxycholic acid paired with newer agents to achieve deeper biochemical response.
- Pruritus treatment trials — Studying IBAT inhibitors and other targeted agents to relieve cholestatic itch.
- Cirrhosis prevention trials — Assessing whether early aggressive treatment delays fibrosis progression in high-risk patients.
- Biomarker studies — Identifying blood markers that predict disease progression and treatment response.
Recently added Primary Biliary Cholangitis trials
Biochemical Response and Clinical Outcomes in Patients With PBC
This study is a bidirectional cohort study. The investigators conduct a bidirectional cohort study utilizing a database in mainland China, continuously collecting demographics, clinical symptoms, and biochemical characteristics of diagnosed PBC patients. The study aims to analyze the association between varying post-treatment alkaline phosphatase (AKP) levels and complication-free survival rates, with the objective to develop and validate a predictive survival model.
A Study to Assess How Well and Safely Elafibranor Works in Adult Participants With Primary Sclerosing Cholangitis
The purpose of this study is to find out how well and safely elafibranor works compared to placebo in adult participants with Primary sclerosing cholangitis (PSC). PSC is a rare disease that causes inflammation and scarring of the bile ducts in the liver. Over time, this can lead to liver damage and serious health problems, including the need for a liver transplant and death. In this study, about 350 participants with large duct PSC will take part. Participants will be randomized to receive either elafibranor 120 mg once daily or a placebo (a tablet with no active medicine). The study includes a screening period, an treatment period, and a post-treatment safety follow-up. During the study, participants will undergo routine clinical assessments, laboratory testing, imaging evaluations, and complete patient-reassessments to evaluate liver disease progression, symptoms, quality of life and safety. Following the end of treatment, participants will complete a safety follow-up period at approximately four weeks. Participants may withdraw from the study at any time. Each participant may be in the study for several years, as the treatment period will continue until the study reaches enough health events among participants, which is expected to take about 5 years.
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