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

Launched by MIRUM PHARMACEUTICALS, INC. · Dec 9, 2020

Keywords

ClinConnect Summary

This clinical trial is studying an investigational drug called volixibat to see how well it helps reduce itching (pruritus) in patients with Primary Sclerosing Cholangitis (PSC), a liver disease. The trial aims to find out not only if volixibat can relieve itching but also if it might have any effect on the progression of PSC itself.

To participate in this study, you should be at least 12 years old (or 18 in some areas) and have a confirmed diagnosis of PSC. You will need to agree to follow all study requirements, including attending visits. If you are currently taking medications for itching or have inflammatory bowel disease, you may still be eligible, as long as you meet other specific criteria. Participants will be monitored throughout the study for safety and effectiveness. This trial is currently recruiting, and all genders are welcome to join. If you're interested, please talk to your healthcare provider for more information.

Gender

ALL

Eligibility 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