A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Biliary Cholangitis
VANTAGE
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
1 other identifier
interventional
333
15 countries
130
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 Biliary Cholangitis (PBC), and to assess the possible impact on the disease progression of PBC.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Sep 2021
Longer than P75 for phase_2
130 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 9, 2021
CompletedFirst Posted
Study publicly available on registry
September 20, 2021
CompletedStudy Start
First participant enrolled
September 22, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 1, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 1, 2028
August 25, 2026
August 1, 2026
5.3 years
September 9, 2021
August 24, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire
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 Outcomes (8)
Proportion of participants with itch response using the Adult ItchRO
Baseline to week 28
Incidence of adverse events
Baseline to week 28
Changes in alkaline phosphatase
Baseline to week 28
Changes in total bilirubin
Baseline to week 28
Changes in serum bile acid levels
Baseline to week 28
- +3 more secondary outcomes
Study Arms (5)
Part 1 Arm 1: Volixibat 20mg
EXPERIMENTALParticipants randomized to this arm will receive volixibat 20mg twice daily.
Part 1 Arm 2: Volixibat 80mg
EXPERIMENTALParticipants randomized to this arm will receive volixibat 80mg twice daily.
Part 1 Arm 3: Placebo
PLACEBO COMPARATORParticipants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
Part 2 Arm 1: Volixibat Selected Dose 20mg
EXPERIMENTALParticipants randomized to this arm will receive volixibat 20mg twice daily.
Part 2 Arm 2: Placebo
PLACEBO COMPARATORParticipants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
Interventions
Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.
Capsules matched to study drug without the active pharmaceutical ingredient
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (130)
Southern California Research Center
Coronado, California, 92218, United States
Cedars Sinai Medical Center
Los Angeles, California, 90048, United States
University of California Los Angeles
Los Angeles, California, 90095, United States
UC Davis
Sacramento, California, 95817, United States
University of Colorado
Aurora, Colorado, 80045, United States
Yale University Digestive Diseases
New Haven, Connecticut, 06510, United States
Covenant Metabolic Specialists
Fort Myers, Florida, 33912, United States
UF Hepatology Research at CTRB
Gainesville, Florida, 32610, United States
UF Health Gastroenterology- Emerson
Jacksonville, Florida, 32209, United States
IHS Health
Kissimmee, Florida, 34741, United States
Schiff Center for Liver Diseases
Miami, Florida, 33136, United States
Advanced Research Institute, Inc
New Port Richey, Florida, 34653, United States
Covenant Research and Clinics, LLC
Sarasota, Florida, 34240, United States
Tampa General Hospital
Tampa, Florida, 33606, United States
Cleveland Clinic Florida
Weston, Florida, 33331, United States
Southeast Clinical Research Center
Dalton, Georgia, 30720, United States
Northwestern University
Chicago, Illinois, 60611, United States
Loyola University Health System - Loyola Outpatient Center
Maywood, Illinois, 60153, United States
University of Iowa Hospitals & Clinics
Iowa City, Iowa, 52242, United States
Ochsner Clinic Foundation-Baton Rouge
Baton Rouge, Louisiana, 70836, United States
LSU Health Science Center Shreveport
Shreveport, Louisiana, 71103, United States
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
Beth Israel Deaconess
Boston, Massachusetts, 02215, United States
Mayo Clinic
Rochester, Minnesota, 55905, United States
Jackson Liver and GI Specialists
Jackson, Mississippi, 39216, United States
CHI Health Clinic (Alegent Creighton Clinic)- Gastroenterology-Bergan
Omaha, Nebraska, 68124, United States
Virtua Center for Liver Disease-Camden
Camden, New Jersey, 08103, United States
Montefiore Medical Center
The Bronx, New York, 10467, United States
Science 37, Inc (Remote-homebased Telemedicine)
Morrisville, North Carolina, 27560, United States
Cleveland Clinic - Main Campus
Cleveland, Ohio, 44195, United States
Einstein Healthcare Network - Einstein Medical Center
Philadelphia, Pennsylvania, 19141, United States
Methodist Le Bonheur Healthcare
Memphis, Tennessee, 38104, United States
Galen Medical Group, P.C
Tennessee City, Tennessee, 37343, United States
Amel Med LLC
Austin, Texas, 78750, United States
The Liver Institute at Methodist Dallas Medical Center
Dallas, Texas, 75203, United States
Soma Clinical Trials
Denison, Texas, 75020, United States
Baylor College of Medicine
Houston, Texas, 77030, United States
Liver Associates of Texas
Houston, Texas, 77030, United States
Impact Research
Waco, Texas, 76710, United States
University of Utah Health Care
Salt Lake City, Utah, 84132, United States
MedStar Health Research Institute
Fairfax, Virginia, 22031, United States
Bon Secours Liver Institute of Hampton Roads Mary Immaculate Hospital
Newport News, Virginia, 23602, United States
Bon Secours Richmond Community Hospital LLC d/b/a Bon Secours Liver Institute of Richmond
Richmond, Virginia, 23226, United States
Liver Institute Northwest
Seattle, Washington, 98105, United States
Hospital de Alta Complejidad En Red El Cruce
San Juan Bautista, Buenos Aires, 1888, Argentina
Hospital Provincial Del Centenario
Rosario, Santa Fe Province, 2000, Argentina
Glenny Corp S.A
CABA, 1430, Argentina
Clínica de Nefrología, Urología y Enfermedades Cardiovasculares
Santa Fe, 3000, Argentina
Universitair Ziekenhuis Antwerpe UZA
Antwerp, 2650, Belgium
Cliniques Universitaires Saint-Luc
Brussels, 1200, Belgium
UZ Gent
Ghent, 9000, Belgium
Centro de Pesquisa Clínica da Bahiana Saúde
Salvador, Estado de Bahia, 40290-000, Brazil
Hospital Das Clínicas Da Ufg
Goiânia, Goiás, 74605-050, Brazil
Centro de Pesquisas Clínicas do HC/UFMG-EBSERH
Belo Horizonte, Minas Gerais, 30130-100, Brazil
Hospital de Clínicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, 90035903, Brazil
Hospital Universitário Clementino Fraga Filho - Secretaria de Hepatologia
Rio de Janeiro, 21941-617, Brazil
Instituto de Educação, Pesquisa e Gestão em Saúde
Rio de Janeiro, 22775-001, Brazil
Fundação Oswaldo Ramos
São Paulo, 04038-002, Brazil
Gordon and Leslie Diamond Health Care Centre, Division of Gastroenterology
Vancouver, British Columbia, V5Z 1M9, Canada
(G.I.R.I.) GI Research Institute Foundation
Vancouver, British Columbia, V6Z 2K5, Canada
McMaster University Medical Center
Hamilton, Ontario, L8S 4K1, Canada
University Hospital, LHSC
London, Ontario, N6A5A5, Canada
Toronto General Hospital
Toronto, Ontario, M5G 1Z5, Canada
McGill University Health Centre
Montreal, Quebec, H4A 3J1, Canada
University of Alberta
Edmonton, T6G 2X8, Canada
CRCHUM (Centre de Recherche du Centre Hospitalier de l'Université de Montréal)
Montreal, H2X 0A9, Canada
Mengchao Hepatobiliary Hospital of Fujian Medical University
Fuzhou, Fujian, 350028, China
Union Hospital Tongji Medical College Huazhong University of Science and Technology
Wuhan, Hubei, 430022, China
The First Hospital of Jilin University
Changchun, Jilin, 130031, China
The First Affiliated Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310003, China
Beijing Youan Hospital, Capital Medical University
Beijing, 100069, China
Beijing Friendship Hospital, Capital Medical University
Beijing, 10050, China
Sir Run Run Shaw Hospital -Zhejiang University School of Medicine
Hangzhou, 310009, China
CHU Grenoble - Hôpital MICHALLON
Grenoble, 38043, France
Hopital Claude Huriez-Digestive system disease clinic
Lille, 59037, France
CHU de NICE- Hospital L'Archet 2
Nice, 06202, France
Hospital Saint Antoine
Paris, 75012, France
Nouvel Hospital Civil
Strasbourg, 67091, France
Hospital Paul Brousse- APHP
Villejuif, 94800, France
Klinikum Chemnitz gGmbH
Chemnitz, 09116, Germany
Universitätsklinikum Frankfurt am Main
Frankfurt, Germany
Medizinische Hochschule Hannover (MHH)
Hanover, 30625, Germany
Universitätsklinikum Schleswig-Holstein
Kiel, 24105, Germany
Universitätsklinikum Leipzig, Klinik und Poliklinik für Onkologie, Gastroenterologie, Hepatologie und Pneumologie
Leipzig, 04103, Germany
Otto-von-Guericke-Universität Klinik für Gastroenterologie, Hepatologie und Infektiologie Universitätsklinikum Magdeburg A.ö.R.
Magdeburg, 39120, Germany
University Hospital Munster
Münster, 48149, Germany
University Hospital Tübingen Medical clinic
Tübingen, 72076, Germany
St. Josefs-Hospital Wiesbaden
Wiesbaden, 65189, Germany
Hillel Yaffe Medical Center
Hadera, 3810101, Israel
Rambam Medical Center
Haifa, 3109601, Israel
Carmel Medical Center
Haifa, 3434104, Israel
Shaare Zedek Medical Center
Jerusalem, 9103102, Israel
Hadassah Medical Center - Ein Karem, Liver Institute Kiryat Hadassah POB 12000
Jerusalem, 9112001, Israel
Galilee Medical Center
Nahariya, 2210001, Israel
Rabin Medical Center
Petah Tikva, 4941492, Israel
Sheba Medical Center
Ramat Gan, 52621, Israel
The Tel Aviv Sourasky Medical Center
Tel Aviv, 6423906, Israel
Universita Politecnica delle Marche- Ospedali Riuniti
Ancona, Italy
ASST Papa Giovanni XXIII
Bergamo, 24127, Italy
Fondazione Ca Granda
Milan, 20122, Italy
ASST Grande Ospedale Metropolitano Niguarda
Milan, 20162, Italy
Azienda Ospedaliero Universitaria Di Modena
Modena, 41126, Italy
Fondazione IRCCS San Gerardo dei Tintori
Monza, 20900, Italy
Azienda Ospedale-Università di Padova
Padova, 35128, Italy
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
Palermo, 90127, Italy
Azienda Ospedaliero Universitaria Pisana (AOUP)
Pisa, 56124, Italy
Istituto Clinico Humanitas
Rozzano, Italy
National Hospital Organization Nagasaki Medical Center
Ōmura, Nagasaki, 856-8562, Japan
Ehime University Hospital
Ehime, 791-0295, Japan
Teine Keijinkai Hospital
Hokkaido, 006-8555, Japan
Osaka Rosai Hospital
Osaka, 591-8025, Japan
Toranomon Hospital
Tokyo, 105-8470, Japan
Amstedam University Medical Centers
Amsterdam, 1081 HV, Netherlands
Radboudumc
Nijmegen, 6525 GA, Netherlands
Erasmus medical center
Rotterdam, Netherlands
Hospital Universitari Vall d' Hebron
Barcelona, 08035, Spain
Hospital Clínic de Barcelona
Barcelona, 08036, Spain
Hospital Universitari Parc Tauli
Barcelona, 08208, Spain
Hospital General Universitario Gregorio Maranon
Madrid, 28007, Spain
Hospital Universitario Virgen del Rocio
Seville, 41013, Spain
Hospital Universitario Miguel Servet
Zaragoza, 50009, Spain
Ente Ospedaliero Cantonale
Lugano, 6900, Switzerland
University Hospital Zurich(USZ)
Zurich, 8091, Switzerland
University Hospital Southampton NHS Foundation Trust
Southampton, Hampshire, S016 6YD, United Kingdom
University Hospitals Birmingham NHS Foundation Trust of Trust Headquarters
Birmingham, West Midlands, B15 2GW, United Kingdom
Belfast Health & Social Care Trust (BHSCT) Royal Victoria Hospital
Belfast, BT12 6BA, United Kingdom
Royal Free Hospital NHS Foundation Trust
London, NW3 2QZ, United Kingdom
King's College Hospital NHS Foundation Trust
London, SE5 9RS, United Kingdom
Freeman Hospital
Newcastle upon Tyne, NE7 7DN, United Kingdom
Nottingham University Hospitals NHS Trust
Nottingham, United Kingdom
Related Publications (1)
Klatzkow H, Bhavsar-Burke I, Pearson M, Wentworth BJ. Primary Biliary Cholangitis in 2025: A New Frontier. Am J Gastroenterol. 2025 Dec 1;120(12):2746-2749. doi: 10.14309/ajg.0000000000003559. Epub 2025 May 29. No abstract available.
PMID: 40439725DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 9, 2021
First Posted
September 20, 2021
Study Start
September 22, 2021
Primary Completion (Estimated)
January 1, 2027
Study Completion (Estimated)
December 1, 2028
Last Updated
August 25, 2026
Record last verified: 2026-08