Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at Sacramento, California and other locations
Dates
study started
estimated completion
Principal Investigator
by Christopher Bowlus, MD

Description

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), and to assess the possible impact on the disease progression of PSC.

Official Title

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

Keywords

Primary Sclerosing Cholangitis Pruritus PSC Itch Itching Cholestasis Cholangitis Cholangitis, Sclerosing Volixibat Volixibat 20mg Volixibat 80mg

Eligibility

You can join if…

Open to people ages 18 years and up

  1. 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.

You CAN'T join if...

  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 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

Locations

  • University of California, Davis accepting new patients
    Sacramento California 95817 United States
  • California Pacific Medical Center Research Institute not yet accepting patients
    San Francisco California 94109 United States

Lead Scientist at UC Davis

  • Christopher Bowlus, MD
    Professor, Gastroenterology and Hepatology. Authored (or co-authored) 180 research publications.

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Mirum Pharmaceuticals, Inc.
Links
Sign up for this study
ID
NCT04663308
Phase
Phase 2
Study Type
Interventional
Last Updated