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Evaluating a new drug for itching in patients with Primary Biliary Cholangitis

A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Volixibat in the Treatment of Cholestatic Pruritus in Patients With Primary Biliary Cholangitis

Phase 2 Interventional Mirum Pharmaceuticals, Inc. · NCT05050136

This study is testing a new drug to see if it can help adults with Primary Biliary Cholangitis who are suffering from itching.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment260 (estimated)
Ages18 Years and up
SexAll
SponsorMirum Pharmaceuticals, Inc. Industry-sponsored
Locations130 sites (Coronado, California and 129 other locations)
Trial IDNCT05050136 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to assess the efficacy and safety of an investigational drug called volixibat in treating pruritus, or itching, associated with Primary Biliary Cholangitis (PBC). Participants will receive either volixibat or a placebo to determine the drug's impact on itching and potential effects on disease progression. The study will include adults aged 18 and older with a confirmed diagnosis of PBC and qualified pruritus. The trial will follow participants through various study visits to monitor their response to the treatment.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18 and older with a confirmed diagnosis of Primary Biliary Cholangitis experiencing pruritus.

Not a fit: Patients with itching caused by conditions other than Primary Biliary Cholangitis will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly alleviate itching for patients suffering from Primary Biliary Cholangitis.

How similar studies have performed: Other studies have explored treatments for pruritus in liver diseases, but the specific approach with volixibat is novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Provide signed informed consent at the screening visit as well as comply with all study visits and requirements through the end of the study
* Male or female, age ≥18 years at the screening visit
* Confirmed diagnosis of PBC in line with the AASLD guidelines
* UDCA, anti-pruritic medication and PPAR agonist use will be allowed if meeting additional criteria
* Qualified pruritus associated with PBC as assessed by Adult ItchRO

Exclusion Criteria:

* Pruritus associated with an etiology other than PBC
* Evidence or clinical suspicion of decompensated cirrhosis or a history of decompensation events
* Current symptomatic cholelithiasis or inflammatory gallbladder disease
* History of small bowel surgery/resection impacting the terminal ileum that may disrupt the enterohepatic circulation.
* Evidence, history, or suspicion of other liver diseases; PBC patients with AIH are not excluded
* History of Liver transplantation

Where this trial is running

Coronado, California and 129 other locations

+80 more sites — see ClinicalTrials.gov for the full list.

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Primary Biliary CholangitisPBCPruritusItchingCholangitisHepatobiliary disorders
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.