A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC)
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.
- Primary Sclerosing Cholangitis
- Eligible Ages
- Over 18 Years
- Eligible Genders
- Accepts Healthy Volunteers
- Provide freely signed informed consent and be willing to comply with all study visits and requirements through end of study, including the follow-up period. 2. Subjects aged ≥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. Qualified 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.
- 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 diseases 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
- Phase 2
- Study Type
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Double (Participant, Investigator)
|Participants randomized to this arm will receive volixibat 20mg twice daily.||
|Participants randomized to this arm will receive volixibat 80mg twice daily.||
|Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.||
- Mirum Pharmaceuticals, Inc.
Study ContactClinical Trials Mirum