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 (VISTAS) - VISTAS

Mirum Pharmaceuticals, Inc.0 个研究点目标入组 300 人开始时间: 2021年6月17日最近更新: 2023年1月9日

适应症

试验速览

暂无简介。

•For screening:
•1. Provide freely signed informed consent and be willing to comply with all study visits and requirements through the end of the long-term extension period.
•2. Age =16 years for eligible regions; otherwise =18 years
•3. Confirmed diagnosis of large duct or small duct PSC based on AASLD guidelines.
•4. Qualified pruritus associated with PSC as assessed by Adult ItchRO
•5. Completion of =80% of daily Adult ItchRO assessments during the screening period
•6. UDCA and anti-pruritic medication use will be allowed if meeting additional criteria. Concomitant IBD is allowed if meeting additional criteria
•7. Participants with AIH who are on stable treatment with medications
•For randomization
•1. Completion of =80% of Adult ItchRO assessments during the single-blind, placebo run- in period
•2. Study drug compliance of =80% during the single-blind, placebo run-in period
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 10
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 170
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 30

•1. Pruritus associated with an etiology other than PSC
•2. Evidence or clinical suspicion of decompensated cirrhosis, or a history of decompensation events
•3. Presumptive or diagnosed ascending cholangitis within 12 weeks of screening through Day 1
•4. Placement of a percutaneous drain or biliary stent within 12 weeks of screening through Day 1
•5. Balloon dilatation procedure of a stricture within 12 weeks of screening through Day 1
•6. History of ileostomy or small bowel surgery/resection or other surgeries that may have disrupted the enterohepatic circulation
•7. ECG with clinically significant abnormalities as determined by the investigator
•8. Evidence, history, or suspicion of other liver and GI-related diseases and malignancies
•9. Positive for HIV antibody
•10. Any active infection that, in the opinion of the investigator or medical monitor, would preclude successful participation in the study
•11. Unstable and/or serious medical disease that is likely to impair the participant’s ability to participate in all aspects of the study, confound efficacy and/or safety assessments, or result in substantially shortened life expectancy
•12. Clinically relevant alcohol use disorder or drug abuse within 24 weeks of screening

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