A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC) (VISTAS)
- Conditions
- Pruritus associated with Primary Sclerosing Cholangitis (PSC)MedDRA version: 20.1Level: LLTClassification code: 10036732Term: Primary sclerosing cholangitis Class: 10019805Therapeutic area: Diseases [C] - Digestive System Diseases [C06]
- Registration Number
- CTIS2023-505764-11-00
- Lead Sponsor
- Mirum Pharmaceuticals Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 395
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., Age =12 years for eligible regions; otherwise =18 years, Have a confirmed PSC diagnosis prior to Visit 2 consistent with the guidelines for the diagnosis and management of PSC published by the AASLD, Average daily Adult ItchRO score =1 during the 2 consecutive weeks leading to Visit 2, 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
Chronic pruritus of other etiology other than that may interfere with the severity assessment of PSC-related pruritus, Evidence or clinical suspicion of decompensated cirrhosis, or a history of decompensation events., Bile duct stent or percutaneous bile duct drain placement, or balloon dilatation procedure of a stricture within 12 weeks of Screening, 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 and GI-related diseases and malignancies., History of liver transplantation, 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
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the efficacy of volixibat versus placebo for the treatment of pruritus (as measured by the change in the Adult ItchRO tool) in participants with PSC.;Secondary Objective: To evaluate the efficacy of volixibat versus placebo on the improvement of markers of cholestasis, To evaluate safety and tolerability of volixibat versus placebo in participants with PSC-associated pruritus., To evaluate the efficacy of volixibat versus placebo for the treatment of pruritus on the basis of proportion of participants achieving a reduction of =2 points in the weekly averaged daily Adult ItchRO score from baseline through Week 24, To evaluate quality of life in participants with PSC-associated pruritus treated with volixibat versus placebo.;Primary end point(s): Mean change in the Adult ItchRO comparing baseline with the average of the weekly averaged worst daily itch scores.
- Secondary Outcome Measures
Name Time Method