A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC)

Running, not enrolling · Phase 2 · Has a placebo group

Conditions studied: Primary Sclerosing Cholangitis

In brief

The purpose of this clinical research study is to learn more about the use of the study medicine, volixibat, for the treatment of pruritus (itching) associated with Primary Sclerosing Cholangitis (PSC), and to assess the possible impact on the disease progression of PSC.

Key facts

Study ID

NCT04663308

Run by

Mirum Pharmaceuticals, Inc.

People needed

182

Starts

2020-12-18

Expected to finish

2027-04-01

Last updated by the study team

2026-02-04

Who can join

Age: 12 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.
• Subjects aged ≥12 years for eligible regions; otherwise ≥18 years
• Confirmed diagnosis of large duct or small duct PSC based on American Association for the Study of Liver Disease (AASLD) guidelines.
• Pruritus associated with PSC as assessed by Adult ItchRO.
• Ursodeoxycholic acid (UDCA) and anti-pruritic medication use will be allowed if meeting additional criteria.
• Concomitant Inflammatory Bowel Disease (IBD) is allowed if meeting additional criteria.

You may not qualify if…

• Pruritus associated with an etiology other than PSC
• Evidence or clinical suspicion of decompensated cirrhosis, or a history of decompensation events
• History of ileostomy or small bowel surgery/resection or other surgeries that may have disrupted the enterohepatic circulation
• Evidence, history, or suspicion of other liver disease; PSC patients with AIH are not excluded.
• Bile duct stent or percutaneous bile duct drain placement, or balloon dilatation procedure of a stricture within 12 weeks of Screening
• 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
• History of liver transplantation

Where it is running

• Cedars Sinai Medical Center — Los Angeles, California, United States
• University of California, Davis — Sacramento, California, United States
• California Pacific Medical Center Research Institute — San Francisco, California, United States
• University of Colorado - Anschutz Medical Campus — Aurora, Colorado, United States
• MedStar Georgetown University Hospital — Washington D.C., District of Columbia, United States
• Florida Research Institute — Lakewood Rch, Florida, United States
• University of Miami - Schiff Center for Liver Diseases — Miami, Florida, United States
• Tampa General Hospital — Tampa, Florida, United States
• Beth Israel Deaconess Medical Center — Boston, Massachusetts, United States
• Henry Ford Hospital — Detroit, Michigan, United States
• Mayo Clinic — Rochester, Minnesota, United States
• Southern Therapy and Advanced Research LLC — Jackson, Mississippi, United States
• Northwell Health — Manhasset, New York, United States
• New York University Langone Health — New York, New York, United States
• Weill Cornell Medical College — New York, New York, United States
• Columbia University Medical Center - Presbyterian Hospital and Vanderbilt Clinic — New York, New York, United States
• University of Rochester Medical Center - Strong Memorial Hospital — Rochester, New York, United States
• Duke Health - Duke University Medical Center — Durham, North Carolina, United States
• Science 37, Inc (Remote-homebased Telemedicine) — Morrisville, North Carolina, United States
• Cleveland Clinic — Cleveland, Ohio, United States
• Einstein Healthcare Network - Einstein Medical Center — Philadelphia, Pennsylvania, United States
• University of Pittsburgh Medical Center — Pittsburgh, Pennsylvania, United States
• Galen Medical Group — Hixson, Tennessee, United States
• Vanderbilt University Medical Center — Nashville, Tennessee, United States
• Southern California Research Center — Coronado, California, United States

Full record on ClinicalTrials.gov

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