Status:
ACTIVE_NOT_RECRUITING
A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC)
Lead Sponsor:
Mirum Pharmaceuticals, Inc.
Conditions:
Primary Sclerosing Cholangitis
Eligibility:
All Genders
12+ years
Phase:
PHASE2
Brief Summary
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...
Eligibility Criteria
Inclusion
- 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.
Exclusion
- 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
Key Trial Info
Start Date :
December 18 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 1 2027
Estimated Enrollment :
182 Patients enrolled
Trial Details
Trial ID
NCT04663308
Start Date
December 18 2020
End Date
April 1 2027
Last Update
November 20 2025
Active Locations (103)
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1
Southern California Research Center
Coronado, California, United States, 92118
2
Cedars Sinai Medical Center
Los Angeles, California, United States, 90048
3
University of California, Davis
Sacramento, California, United States, 95817
4
California Pacific Medical Center Research Institute
San Francisco, California, United States, 94109