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

Phase 2

Recruiting

• Conditions: Primary Sclerosing Cholangitis
• Interventions: Drug: Volixibat; Drug: Placebo

• Registration Number: NCT04663308
• Lead Sponsor: Mirum Pharmaceuticals, Inc.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-11-17
• Study First Submitted QC: 2020-12-09
• Study First Posted: 2020-12-11
• Study Start: 2020-12-18
• Status Verified: 2025-08
• Last Update Submitted: 2025-08-21
• Last Update Posted: 2025-08-24
• Primary Completion: 2025-09
• Study Completion: 2025-12-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 200

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Part 1 Arm 1: Volixibat 20mg	Volixibat	Participants randomized to this arm will receive volixibat 20mg twice daily.
Part 1 Arm 3: Placebo	Placebo	Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
Part 2 Arm 2: Placebo	Placebo	Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
Part 1 Arm 2: Volixibat 80mg	Volixibat	Participants randomized to this arm will receive volixibat 80mg twice daily.
Part 2 Arm 1: Volixibat Selected Dose 20mg	Volixibat	Participants randomized to this arm will receive volixibat 20mg twice daily.

Primary Outcome Measures

Name	Time	Method
Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire	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 Outcome Measures

Name	Time	Method
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	The PROMIS® Fatigue questionnaire is a 5-point response scale. Respondents endorse each item using a 5-point response scale: Never, Rarely, Sometimes, Often, and Always.
Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) sleep disturbance questionnaire	Baseline through to Week 28	The PROMIS® Sleep Disturbance in an 8-item measure that assesses quality of sleep, sleep depth, and restoration associated with sleep. It is a 5-point response scale, the specific response options vary by items. The first 4 items use response options that range from Not at all to Very much. The next 3 items use response options that range from Never to Always. The final item assesses sleep quality ranging from Very poor to Very good.

Trial Locations

Locations (103)

Southern California Research Center; 🇺🇸 Coronado, California, United States

Cedars Sinai Medical Center; 🇺🇸 Los Angeles, California, United States

University of California, Davis; 🇺🇸 Sacramento, California, United States

California Pacific Medical Center Research Institute; 🇺🇸 San Francisco, California, United States

University of Colorado - Anschutz Medical Campus; 🇺🇸 Aurora, Colorado, United States

MedStar Georgetown University Hospital; 🇺🇸 Washington, District of Columbia, United States

Florida Research Institute; 🇺🇸 Lakewood Ranch, Florida, United States

University of Miami - Schiff Center for Liver Diseases; 🇺🇸 Miami, Florida, United States

Tampa General Hospital; 🇺🇸 Tampa, Florida, United States

Beth Israel Deaconess Medical Center; 🇺🇸 Boston, Massachusetts, United States

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