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

Phase 2 Interventional Mirum Pharmaceuticals, Inc. · NCT04663308

This study is testing a new drug called volixibat to see if it can help relieve itching in people with Primary Sclerosing Cholangitis.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment200 (estimated)
Ages12 Years and up
SexAll
SponsorMirum Pharmaceuticals, Inc. Industry-sponsored
Locations103 sites (Coronado, California and 102 other locations)
Trial IDNCT04663308 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to assess the efficacy and safety of an investigational drug named volixibat in treating pruritus (itching) associated with Primary Sclerosing Cholangitis (PSC). Participants will receive either volixibat or a placebo, and the study will monitor the impact on itching and potential disease progression. The trial includes patients aged 12 and older with a confirmed diagnosis of PSC and allows for the use of certain medications during the study. The goal is to provide insights into a new treatment option for this challenging symptom.

Who should consider this trial

Good fit: Ideal candidates include individuals aged 12 years and older with a confirmed diagnosis of Primary Sclerosing Cholangitis experiencing associated pruritus.

Not a fit: Patients whose itching is caused by conditions other than PSC will not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new therapeutic option for patients suffering from severe itching due to PSC.

How similar studies have performed: While there have been studies on pruritus treatments, the specific approach with volixibat in PSC is novel and has not been extensively tested.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

1. Provide freely signed informed consent and assent (as applicable) and be willing to comply with all study visits and requirements through end of study, including the follow-up period.
2. Subjects aged ≥12 years for eligible regions; otherwise ≥18 years
3. Confirmed diagnosis of large duct or small duct PSC based on American Association for the Study of Liver Disease (AASLD) guidelines.
4. Pruritus associated with PSC as assessed by Adult ItchRO.
5. Ursodeoxycholic acid (UDCA) and anti-pruritic medication use will be allowed if meeting additional criteria.
6. Concomitant Inflammatory Bowel Disease (IBD) is allowed if meeting additional criteria.

Exclusion Criteria:

1. Pruritus associated with an etiology other than PSC
2. Evidence or clinical suspicion of decompensated cirrhosis, or a history of decompensation events
3. History of ileostomy or small bowel surgery/resection or other surgeries that may have disrupted the enterohepatic circulation
4. Evidence, history, or suspicion of other liver disease; PSC patients with AIH are not excluded.
5. Bile duct stent or percutaneous bile duct drain placement, or balloon dilatation procedure of a stricture within 12 weeks of Screening
6. Exceeding pre-defined biochemical values for alanine aminotransferase/aspartate aminotransferase (ALT/AST), estimated glomerular filtration rate (eGFR),serum creatinine (sCr), platelet count, international normalized ratio (INR) and total bilirubin
7. History of liver transplantation

Where this trial is running

Coronado, California and 102 other locations

+53 more sites — see ClinicalTrials.gov for the full list.

Study contacts

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 Sclerosing CholangitisPruritusPSCItchItchingCholestasis
Last reviewed 2026-06-14 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.