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

April 23, 2024 updated by: Mirum Pharmaceuticals, Inc.

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 Biliary Cholangitis

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

180

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Grenoble, France, 38043
        • Recruiting
        • CHU GRENOBLE - Hopital Michallon
        • Contact:
      • Lille, France, 59037
        • Recruiting
        • Hopital Claude Huriez-Digestive system disease clinic
        • Contact:
      • Nice, France, 06202
        • Recruiting
        • CHU de NICE- Hospital L'Archet 2
        • Contact:
      • Paris, France, 75012
        • Recruiting
        • Hospital Saint Antoine
        • Contact:
      • Strasbourg, France, 67091
      • Villejuif, France, 94800
  • Germany
      • Chemnitz, Germany, 09116
        • Recruiting
        • Klinikum Chemnitz gGmbH
        • Contact:
          • Jacqueline Uhle
          • Phone Number: +49 (0)371-333 35631
          • Email: juhle@skc.de
        • Contact:
          • Phone Number: +49 (0)371-333 33236
      • Frankfurt, Germany
        • Recruiting
        • Universitätsklinikum Frankfurt am Main
        • Contact:
      • Hanover, Germany, 30625
      • Kiel, Germany, 24105
        • Recruiting
        • Universitatsklinikum Schleswig-Holstein
        • Contact:
        • Contact:
      • Münster, Germany, 48149
      • Tubingen, Germany, 72076
      • Wiesbaden, Germany, 65189
        • Recruiting
        • St. Josefs-Hospital Wiesbaden
        • Contact:
  • Israel
      • Hadera, Israel, 3810101
        • Recruiting
        • Hillel Yaffe Medical Center
        • Contact:
      • Haifa, Israel, 3109601
      • Haifa, Israel, 3434104
      • Jerusalem, Israel, 9112001
        • Recruiting
        • Hadassah Medical Center - Ein Karem, Liver Institute Kiryat Hadassah POB 12000
        • Contact:
        • Contact:
      • Jerusalem, Israel, 9103102
        • Terminated
        • Shaare Zedek Medical Center
      • Nahariyya, Israel, 2210001
        • Recruiting
        • Galilee Medical Center
        • Contact:
      • Petach Tikva, Israel, 4941492
        • Recruiting
        • Rabin Medical Center
        • Contact:
      • Ramat Gan, Israel, 52621
      • Tel Aviv, Israel, 6423906
        • Terminated
        • The Tel Aviv Sourasky Medical Center
  • Italy
      • Ancona, Italy
        • Recruiting
        • Universita Politecnica delle Marche- Ospedali Riuniti
        • Contact:
      • Milano, Italy, 20162
      • Milano, Italy, 20122
      • Monza, Italy, 20900
      • Padova, Italy, 35128
        • Recruiting
        • Azienda Ospedale-Università di Padova
        • Contact:
      • Pisa, Italy, 56124
        • Recruiting
        • Azienda Ospedaliero Universitaria Pisana (AOUP)
        • Contact:
      • Rozzano, Italy
  • United Kingdom
      • Belfast, United Kingdom, BT12 6BA
      • London, United Kingdom, SE5 9RS
        • Recruiting
        • King's College Hospital NHS Foundation Trust
        • Contact:
      • Nottingham, United Kingdom
        • Recruiting
        • Nottingham University Hospitals Nhs Trust
        • Contact:
  • United States
    • California
      • Coronado, California, United States, 92218
        • Terminated
        • Southern California Research Center
      • Culver City, California, United States, 90230
        • Recruiting
        • Science 37, Inc (Remote-homebased Telemedicine)
        • Contact:
      • Los Angeles, California, United States, 90048
    • Colorado
      • Aurora, Colorado, United States, 80045
    • Florida
      • Fort Myers, Florida, United States, 33912
      • Gainesville, Florida, United States, 32610
      • Jacksonville, Florida, United States, 32209
      • Kissimmee, Florida, United States, 34741
      • Miami, Florida, United States, 33136
        • Recruiting
        • Schiff Center for Liver Diseases
        • Contact:
      • New Port Richey, Florida, United States, 34653
      • Sarasota, Florida, United States, 34240
      • Tampa, Florida, United States, 33606
        • Recruiting
        • Tampa General Hospital
        • Contact:
    • Georgia
      • Dalton, Georgia, United States, 30720
    • Illinois
      • Chicago, Illinois, United States, 60611
      • Maywood, Illinois, United States, 60153
        • Recruiting
        • Loyola University Health System - Loyola Outpatient Center
        • Contact:
        • Contact:
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • Recruiting
        • University of Iowa Hospitals & Clinics
        • Contact:
    • Louisiana
      • Baton Rouge, Louisiana, United States, 70836
        • Recruiting
        • Ochsner Clinic Foundation-Baton Rouge
        • Contact:
      • Shreveport, Louisiana, United States, 71103
        • Recruiting
        • LSU Health Science Center Shreveport
        • Contact:
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
    • Minnesota
      • Rochester, Minnesota, United States, 55905
    • Mississippi
      • Jackson, Mississippi, United States, 39216
        • Terminated
        • Jackson Liver and GI Specialists
    • Nebraska
      • Omaha, Nebraska, United States, 68124
        • Recruiting
        • CHI Health Clinic (Alegent Creighton Clinic)- Gastroenterology-Bergan
        • Contact:
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Recruiting
        • Cleveland Clinic - Main Campus
        • Contact:
        • Contact:
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19141
        • Recruiting
        • Einstein Healthcare Network - Einstein Medical Center
        • Contact:
    • Tennessee
      • Memphis, Tennessee, United States, 38104
        • Terminated
        • Methodist Le Bonheur Healthcare
      • Tennessee, Tennessee, United States, 37343
        • Recruiting
        • Galen Medical Group, P.C
        • Contact:
          • Sydney Boles
          • Phone Number: 423-954-9015
        • Contact:
    • Texas
      • Austin, Texas, United States, 78750
        • Recruiting
        • Amel Med LLC
        • Contact:
      • Denison, Texas, United States, 75020
      • Houston, Texas, United States, 77030
        • Recruiting
        • Liver Associates of Texas
        • Contact:
      • Waco, Texas, United States, 76710
    • Utah
      • Salt Lake City, Utah, United States, 84132
        • Recruiting
        • University of Utah Health Care
        • Contact:
    • Virginia
      • Fairfax, Virginia, United States, 22031
        • Recruiting
        • Medstar Health Research Institute
        • Contact:
      • Newport News, Virginia, United States, 23602
        • Recruiting
        • Bon Secours Liver Institute of Hampton Roads Mary Immaculate Hospital
        • Contact:
      • Richmond, Virginia, United States, 23226
        • Recruiting
        • Bon Secours Richmond Community Hospital LLC d/b/a Bon Secours Liver Institute of Richmond
        • Contact:
    • Washington
      • Seattle, Washington, United States, 98105

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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 and anti-pruritic medication 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Volixibat 20mg
Participants randomized to this arm will receive volixibat 20mg twice daily.
Drug: Volixibat
Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.
Other Names:
  • SHP626
Experimental: Volixibat 80mg
Participants randomized to this arm will receive volixibat 80mg twice daily.
Drug: Volixibat
Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.
Other Names:
  • SHP626
Placebo Comparator: Placebo
Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
Drug: Placebo
Capsules matched to study drug without the active pharmaceutical ingredient

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire
Time Frame: 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.
Baseline to week 28

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Proportion of participants with itch response using the Adult ItchRO
Time Frame: Baseline to week 28
Baseline to week 28
Incidence of adverse events
Time Frame: Baseline to week 28
Baseline to week 28
Changes in alkaline phosphatase
Time Frame: Baseline to week 28
Baseline to week 28
Changes in total bilirubin
Time Frame: Baseline to week 28
Baseline to week 28
Change in Primary Biliary Cholangitis Quality of Life Measure (PBC-40)
Time Frame: Baseline to week 28
PBC-40 questionnaire is scored on a scale of 40 to 200, with higher scores indicating poorer quality of life
Baseline to week 28
Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) fatigue questionnaire
Time Frame: Baseline 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.
Baseline to week 28
Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) sleep disturbance questionnaire
Time Frame: Baseline 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.
Baseline to week 28
Changes in serum bile acid levels
Time Frame: Baseline to week 28
Baseline to week 28

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Mirum Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 22, 2021

Primary Completion (Estimated)

December 1, 2025

Study Completion (Estimated)

December 1, 2025

Study Registration Dates

First Submitted

September 9, 2021

First Submitted That Met QC Criteria

September 10, 2021

First Posted (Actual)

September 20, 2021

Study Record Updates

Last Update Posted (Actual)

April 25, 2024

Last Update Submitted That Met QC Criteria

April 23, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VLX-601

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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