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
Biodegradable Stents in Primary Sclerosing Cholangitis
In patients with PSC, endoscopic therapy of strictures aims to improve cholestasis by relieving the biliary obstruction via endoscopic biliary dilatation with consideration of plastic stents in strictures refractory to dilatation due to the risk of pancreatitis and cholangitis . Short term stents have been shown to have similar recurrence-free rates compared to dilatation in a randomised control trial; however, this was terminated after interim analysis due to higher rates of serious adverse events in the stent group. The long term benefits are unclear; however, it may lead to improved survival compared to predicted survival. In this group of patients with limited treatment options, biodegradable stents may provide an attractive additional treatment modality in the management of high grade strictures.
Austrian PBC Registry
The goal of this registry is to better understand how primary biliary cholangitis develops over time, including the role of disease-related biomarkers, complications of the disease, and symptom burden. Patients with primary biliary cholangitis treated at participating centres in Austria will be invited to take part in this prospective registry. Participation in an associated biobank is optional. Clinical and laboratory data will be collected, and patients will be followed regularly through scheduled clinic visits. In addition, biological samples (serum, plasma, and, if available, liver tissue) may be collected and stored in the biobank for future research.
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