NCT04663308
Active, not recruiting
Phase 2
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
Overview
- Phase
- Phase 2
- Intervention
- Volixibat
- Conditions
- Primary Sclerosing Cholangitis
- Sponsor
- Mirum Pharmaceuticals, Inc.
- Enrollment
- 182
- Locations
- 156
- Primary Endpoint
- Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire
- Status
- Active, not recruiting
- Last Updated
- 2 months ago
Overview
Brief 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.
Investigators
Eligibility Criteria
Inclusion Criteria
- •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.
- •Subjects aged ≥12 years for eligible regions; otherwise ≥18 years
- •Confirmed diagnosis of large duct or small duct PSC based on American Association for the Study of Liver Disease (AASLD) guidelines.
- •Pruritus associated with PSC as assessed by Adult ItchRO.
- •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.
Exclusion Criteria
- •Pruritus associated with an etiology other than PSC
- •Evidence or clinical suspicion of decompensated cirrhosis, or a history of decompensation events
- •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 disease; PSC patients with AIH are not excluded.
- •Bile duct stent or percutaneous bile duct drain placement, or balloon dilatation procedure of a stricture within 12 weeks of Screening
- •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
- •History of liver transplantation
Arms & Interventions
Part 1 Arm 1: Volixibat 20mg
Participants randomized to this arm will receive volixibat 20mg twice daily.
Intervention: Volixibat
Part 1 Arm 2: Volixibat 80mg
Participants randomized to this arm will receive volixibat 80mg twice daily.
Intervention: Volixibat
Part 1 Arm 3: Placebo
Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
Intervention: Placebo
Part 2 Arm 1: Volixibat Selected Dose 20mg
Participants randomized to this arm will receive volixibat 20mg twice daily.
Intervention: Volixibat
Part 2 Arm 2: Placebo
Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
Intervention: Placebo
Outcomes
Primary Outcomes
Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire
Time Frame: Baseline through to Week 28
The Adult ItchRO is an 11-point NRS measurement of itch severity ranging from 0=no itch to 10=worst possible itch.
Secondary Outcomes
- The incidence of adverse events(Baseline through to Week 28)
- Proportion of participants with itch response using the Adult ItchRO(Baseline through to Week 28)
- Changes in serum bile acid levels(Baseline through to Week 28)
- Changes in alkaline phosphatase(Baseline through to Week 28)
- Changes in total bilirubin levels(Baseline through to Week 28)
- Change in Primary Sclerosing Cholangitis-Specific Patient-Reported Outcome(Baseline through to Week 28)
- Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) fatigue questionnaire(Baseline through to Week 28)
- Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) sleep disturbance questionnaire(Baseline through to Week 28)
Study Sites (156)
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Related News
Mirum Pharmaceuticals Completes Enrollment in Phase 2b VISTAS Study for Primary Sclerosing Cholangitis Treatment- Mirum Pharmaceuticals has completed enrollment in the Phase 2b VISTAS study evaluating volixibat, an oral IBAT inhibitor, for treating cholestatic pruritus in primary sclerosing cholangitis patients.
- The trial previously met pre-specified efficacy and safety thresholds at a blinded interim analysis in 2024, with topline results expected in the second quarter of 2026.
- Primary sclerosing cholangitis is a rare, progressive liver disease with no currently approved therapies, leaving patients with limited treatment options focused on symptom management.
- Volixibat works by selectively inhibiting the ileal bile acid transporter to reduce bile acid recycling and has received breakthrough therapy designation for cholestatic pruritus in primary biliary cholangitis.Phase 3 Trial Evaluating Maralixibat for Cholestasis-Related Pruritus- A Phase 3 clinical trial (EXPAND) is underway to assess maralixibat's efficacy in treating pruritus associated with rare cholestatic conditions, excluding Alagille syndrome and PFIC.
- The EXPAND trial aims to enroll 90 participants aged 6 months and older across multiple sites in the U.S., South America, Europe, and the Middle East, with enrollment expected to conclude in 2026.
- Participants will receive either maralixibat or a placebo, with the primary goal of assessing changes in the caregiver-reported ItchRO(Obs) severity score.
- Mirum Pharmaceuticals is also investigating volixibat for pruritus in primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), with Phase 2 trial data expected in 2026.