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

NCT05050136 · tracked via the Priya Life Science UK tracker

Sponsor

Mirum Pharmaceuticals, Inc.

Phase

Phase 2

Started

2021-09-22

Last updated

2026-08-25

Condition(s) studied

Primary Biliary CholangitisPBC

Investigational drug(s) / intervention(s)

VolixibatPlacebo

Volixibat: Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.

Placebo: Capsules matched to study drug without the active pharmaceutical ingredient

Study 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 Biliary Cholangitis (PBC), and to assess the possible impact on the disease progression of PBC.

Eligibility

Sex

ALL

Min age

18 Years

Max age

—

Healthy volunteers

Inclusion Criteria: * Provide signed informed consent at the screening visit as well as comply with all study visits and requirements through the end of the study * Male or female, age ≥18 years at the screening visit * Confirmed diagnosis of PBC in line with the AASLD guidelines * UDCA, anti-pruritic medication and PPAR agonist use will be allowed if meeting additional criteria * Qualified pruritus associated with PBC as assessed by Adult ItchRO Exclusion Criteria: * Pruritus associated with an etiology other than PBC * Evidence or clinical suspicion of decompensated cirrhosis or a history of decompensation events * Current symptomatic cholelithiasis or inflammatory gallbladder disease * History of small bowel surgery/resection impacting the terminal ileum that may disrupt the enterohepatic circulation. * Evidence, history, or suspicion of other liver diseases; PBC patients with AIH are not excluded * History of Liver transplantation

Primary outcome measure(s)

Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire — Baseline 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.

Trial sites (130)

Facility	City	Region
Southern California Research Center	Coronado	California
Cedars Sinai Medical Center	Los Angeles	California
University of California Los Angeles	Los Angeles	California
UC Davis	Sacramento	California
University of Colorado	Aurora	Colorado
Yale University Digestive Diseases	New Haven	Connecticut
Covenant Metabolic Specialists	Fort Myers	Florida
UF Hepatology Research at CTRB	Gainesville	Florida
UF Health Gastroenterology- Emerson	Jacksonville	Florida
IHS Health	Kissimmee	Florida
Schiff Center for Liver Diseases	Miami	Florida
Advanced Research Institute, Inc	New Port Richey	Florida
Covenant Research and Clinics, LLC	Sarasota	Florida
Tampa General Hospital	Tampa	Florida
Cleveland Clinic Florida	Weston	Florida
Southeast Clinical Research Center	Dalton	Georgia
Northwestern University	Chicago	Illinois
Loyola University Health System - Loyola Outpatient Center	Maywood	Illinois
University of Iowa Hospitals & Clinics	Iowa City	Iowa
Ochsner Clinic Foundation-Baton Rouge	Baton Rouge	Louisiana
LSU Health Science Center Shreveport	Shreveport	Louisiana
Massachusetts General Hospital	Boston	Massachusetts
Beth Israel Deaconess	Boston	Massachusetts
Mayo Clinic	Rochester	Minnesota
Jackson Liver and GI Specialists	Jackson	Mississippi
CHI Health Clinic (Alegent Creighton Clinic)- Gastroenterology-Bergan	Omaha	Nebraska
Virtua Center for Liver Disease-Camden	Camden	New Jersey
Montefiore Medical Center	The Bronx	New York
Science 37, Inc (Remote-homebased Telemedicine)	Morrisville	North Carolina
Cleveland Clinic - Main Campus	Cleveland	Ohio
Einstein Healthcare Network - Einstein Medical Center	Philadelphia	Pennsylvania
Methodist Le Bonheur Healthcare	Memphis	Tennessee
Galen Medical Group, P.C	Tennessee City	Tennessee
Amel Med LLC	Austin	Texas
The Liver Institute at Methodist Dallas Medical Center	Dallas	Texas
Soma Clinical Trials	Denison	Texas
Baylor College of Medicine	Houston	Texas
Liver Associates of Texas	Houston	Texas
Impact Research	Waco	Texas
University of Utah Health Care	Salt Lake City	Utah

+ 90 more sites — see the full list on the official registry below.

More Mirum Pharmaceuticals, Inc. trials in the UK

Study to Evaluate Switching to Brelovitug for the Treatment of CHD in Participants Receiving Bulevirtide Recruiting

A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC) Active, not recruiting

Evaluation of Maralixibat in Pruritus Associated With General Cholestatic Liver Disease (EXPAND) Active, not recruiting

A Trial Evaluating Brelovitug (BJT-778) vs Bulevirtide for the Treatment of Chronic Hepatitis Delta Infection (AZURE-2) Recruiting

Other trials for the same condition

A Study Observing Everyday Effectiveness and Safety of the Drug Elafibranor in Participants With Primary Biliary Cholangitis Who Are Receiving Ongoing Treatment Recruiting

Seladelpar in Subjects With Primary Biliary Cholangitis (PBC) and Compensated Cirrhosis Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05050136 on ClinicalTrials.gov ↗ ← All trials in the UK