Primary Biliary CholangitisPhase 2VANTAGENCT05050136
A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Biliary Cholangitis
A Phase 2 study testing Volixibat in people with Primary Biliary Cholangitis and PBC, run by Mirum Pharmaceuticals, Inc. at 130 sites in 15 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Biliary Cholangitis, PBC
- What is being tested?
- Volixibat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 333 participants
- Where
- 130 sitesin 15 countries
- Study dates
- 22 Sep 2021to Dec 2028
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
130 sites in 15 countries.
130 sites in 15 countries
United States44 sites
University of Colorado
Aurora, Colorado, 80045
Amel Med LLC
Austin, Texas, 78750
Ochsner Clinic Foundation-Baton Rouge
Baton Rouge, Louisiana, 70836
Massachusetts General Hospital
Boston, Massachusetts, 02114
Beth Israel Deaconess
Boston, Massachusetts, 02215
Virtua Center for Liver Disease-Camden
Camden, New Jersey, 08103
Northwestern University
Chicago, Illinois, 60611
Cleveland Clinic - Main Campus
Cleveland, Ohio, 44195
Southern California Research Center
Coronado, California, 92218
The Liver Institute at Methodist Dallas Medical Center
Dallas, Texas, 75203
Southeast Clinical Research Center
Dalton, Georgia, 30720
Soma Clinical Trials
Denison, Texas, 75020
MedStar Health Research Institute
Fairfax, Virginia, 22031
Covenant Metabolic Specialists
Fort Myers, Florida, 33912
UF Hepatology Research at CTRB
Gainesville, Florida, 32610
Baylor College of Medicine
Houston, Texas, 77030
Liver Associates of Texas
Houston, Texas, 77030
University of Iowa Hospitals & Clinics
Iowa City, Iowa, 52242
Jackson Liver and GI Specialists
Jackson, Mississippi, 39216
UF Health Gastroenterology- Emerson
Jacksonville, Florida, 32209
IHS Health
Kissimmee, Florida, 34741
Cedars Sinai Medical Center
Los Angeles, California, 90048
University of California Los Angeles
Los Angeles, California, 90095
Loyola University Health System - Loyola Outpatient Center
Maywood, Illinois, 60153
Methodist Le Bonheur Healthcare
Memphis, Tennessee, 38104
Schiff Center for Liver Diseases
Miami, Florida, 33136
Science 37, Inc (Remote-homebased Telemedicine)
Morrisville, North Carolina, 27560
Yale University Digestive Diseases
New Haven, Connecticut, 06510
Advanced Research Institute, Inc
New Port Richey, Florida, 34653
Bon Secours Liver Institute of Hampton Roads Mary Immaculate Hospital
Newport News, Virginia, 23602
CHI Health Clinic (Alegent Creighton Clinic)- Gastroenterology-Bergan
Omaha, Nebraska, 68124
Einstein Healthcare Network - Einstein Medical Center
Philadelphia, Pennsylvania, 19141
Bon Secours Richmond Community Hospital LLC d/b/a Bon Secours Liver Institute of Richmond
Richmond, Virginia, 23226
Mayo Clinic
Rochester, Minnesota, 55905
UC Davis
Sacramento, California, 95817
University of Utah Health Care
Salt Lake City, Utah, 84132
Covenant Research and Clinics, LLC
Sarasota, Florida, 34240
Liver Institute Northwest
Seattle, Washington, 98105
LSU Health Science Center Shreveport
Shreveport, Louisiana, 71103
Tampa General Hospital
Tampa, Florida, 33606
Galen Medical Group, P.C
Tennessee City, Tennessee, 37343
Montefiore Medical Center
The Bronx, New York, 10467
Impact Research
Waco, Texas, 76710
Cleveland Clinic Florida
Weston, Florida, 33331
Italy10 sites
Universita Politecnica delle Marche- Ospedali Riuniti
Ancona
ASST Papa Giovanni XXIII
Bergamo, 24127
Fondazione Ca Granda
Milan, 20122
ASST Grande Ospedale Metropolitano Niguarda
Milan, 20162
Azienda Ospedaliero Universitaria Di Modena
Modena, 41126
Fondazione IRCCS San Gerardo dei Tintori
Monza, 20900
Azienda Ospedale-Università di Padova
Padova, 35128
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
Palermo, 90127
Azienda Ospedaliero Universitaria Pisana (AOUP)
Pisa, 56124
Istituto Clinico Humanitas
Rozzano
Germany9 sites
Klinikum Chemnitz gGmbH
Chemnitz, 09116
Universitätsklinikum Frankfurt am Main
Frankfurt
Medizinische Hochschule Hannover (MHH)
Hanover, 30625
Universitätsklinikum Schleswig-Holstein
Kiel, 24105
Universitätsklinikum Leipzig, Klinik und Poliklinik für Onkologie, Gastroenterologie, Hepatologie und Pneumologie
Leipzig, 04103
Otto-von-Guericke-Universität Klinik für Gastroenterologie, Hepatologie und Infektiologie Universitätsklinikum Magdeburg A.ö.R.
Magdeburg, 39120
University Hospital Munster
Münster, 48149
University Hospital Tübingen Medical clinic
Tübingen, 72076
St. Josefs-Hospital Wiesbaden
Wiesbaden, 65189
Israel9 sites
Hillel Yaffe Medical Center
Hadera, 3810101
Rambam Medical Center
Haifa, 3109601
Carmel Medical Center
Haifa, 3434104
Shaare Zedek Medical Center
Jerusalem, 9103102
Hadassah Medical Center - Ein Karem, Liver Institute Kiryat Hadassah POB 12000
Jerusalem, 9112001
Galilee Medical Center
Nahariya, 2210001
Rabin Medical Center
Petah Tikva, 4941492
Sheba Medical Center
Ramat Gan, 52621
The Tel Aviv Sourasky Medical Center
Tel Aviv, 6423906
Canada8 sites
University of Alberta
Edmonton, T6G 2X8
McMaster University Medical Center
Hamilton, Ontario, L8S 4K1
University Hospital, LHSC
London, Ontario, N6A5A5
McGill University Health Centre
Montreal, Quebec, H4A 3J1
CRCHUM (Centre de Recherche du Centre Hospitalier de l'Université de Montréal)
Montreal, H2X 0A9
Toronto General Hospital
Toronto, Ontario, M5G 1Z5
Gordon and Leslie Diamond Health Care Centre, Division of Gastroenterology
Vancouver, British Columbia, V5Z 1M9
(G.I.R.I.) GI Research Institute Foundation
Vancouver, British Columbia, V6Z 2K5
Brazil7 sites
Centro de Pesquisas Clínicas do HC/UFMG-EBSERH
Belo Horizonte, Minas Gerais, 30130-100
Hospital Das Clínicas Da Ufg
Goiânia, Goiás, 74605-050
Hospital de Clínicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, 90035903
Hospital Universitário Clementino Fraga Filho - Secretaria de Hepatologia
Rio de Janeiro, 21941-617
Instituto de Educação, Pesquisa e Gestão em Saúde
Rio de Janeiro, 22775-001
Centro de Pesquisa Clínica da Bahiana Saúde
Salvador, Estado de Bahia, 40290-000
Fundação Oswaldo Ramos
São Paulo, 04038-002
China7 sites
Beijing Youan Hospital, Capital Medical University
Beijing, 100069
Beijing Friendship Hospital, Capital Medical University
Beijing, 10050
The First Hospital of Jilin University
Changchun, Jilin, 130031
Mengchao Hepatobiliary Hospital of Fujian Medical University
Fuzhou, Fujian, 350028
The First Affiliated Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310003
Sir Run Run Shaw Hospital -Zhejiang University School of Medicine
Hangzhou, 310009
Union Hospital Tongji Medical College Huazhong University of Science and Technology
Wuhan, Hubei, 430022
United Kingdom7 sites
Belfast Health & Social Care Trust (BHSCT) Royal Victoria Hospital
Belfast, BT12 6BA
University Hospitals Birmingham NHS Foundation Trust of Trust Headquarters
Birmingham, West Midlands, B15 2GW
Royal Free Hospital NHS Foundation Trust
London, NW3 2QZ
King's College Hospital NHS Foundation Trust
London, SE5 9RS
Freeman Hospital
Newcastle upon Tyne, NE7 7DN
Nottingham University Hospitals NHS Trust
Nottingham
University Hospital Southampton NHS Foundation Trust
Southampton, Hampshire, S016 6YD
France6 sites
CHU Grenoble - Hôpital MICHALLON
Grenoble, 38043
Hopital Claude Huriez-Digestive system disease clinic
Lille, 59037
CHU de NICE- Hospital L'Archet 2
Nice, 06202
Hospital Saint Antoine
Paris, 75012
Nouvel Hospital Civil
Strasbourg, 67091
Hospital Paul Brousse- APHP
Villejuif, 94800
Spain6 sites
Hospital Universitari Vall d' Hebron
Barcelona, 08035
Hospital Clínic de Barcelona
Barcelona, 08036
Hospital Universitari Parc Tauli
Barcelona, 08208
Hospital General Universitario Gregorio Maranon
Madrid, 28007
Hospital Universitario Virgen del Rocio
Seville, 41013
Hospital Universitario Miguel Servet
Zaragoza, 50009
Japan5 sites
Ehime University Hospital
Ehime, 791-0295
Teine Keijinkai Hospital
Hokkaido, 006-8555
National Hospital Organization Nagasaki Medical Center
Ōmura, Nagasaki, 856-8562
Osaka Rosai Hospital
Osaka, 591-8025
Toranomon Hospital
Tokyo, 105-8470
Argentina4 sites
Glenny Corp S.A
CABA, 1430
Hospital Provincial Del Centenario
Rosario, Santa Fe Province, 2000
Hospital de Alta Complejidad En Red El Cruce
San Juan Bautista, Buenos Aires, 1888
Clínica de Nefrología, Urología y Enfermedades Cardiovasculares
Santa Fe, 3000
Belgium3 sites
Universitair Ziekenhuis Antwerpe UZA
Antwerp, 2650
Cliniques Universitaires Saint-Luc
Brussels, 1200
UZ Gent
Ghent, 9000
Netherlands3 sites
Amstedam University Medical Centers
Amsterdam, 1081 HV
Radboudumc
Nijmegen, 6525 GA
Erasmus medical center
Rotterdam
Switzerland2 sites
Ente Ospedaliero Cantonale
Lugano, 6900
University Hospital Zurich(USZ)
Zurich, 8091
About this study
Official title: 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.
- Conditions
- Primary Biliary Cholangitis, PBC
- Being tested
- Volixibat, Placebo
- Keywords
- Pruritus, PBC, Itching, Cholangitis, Primary Biliary Cholangitis, Hepatobiliary disorders
- Medical subject headings
- Liver Cirrhosis, Biliary, Pruritus, Cholangitis, Digestive System Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VolixibatDrug
Also called SHP626
Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.
PlaceboDrug
Capsules matched to study drug without the active pharmaceutical ingredient
| Study group | Role | What they receive |
|---|---|---|
| Part 1 Arm 1: Volixibat 20mg | Experimental | Participants randomized to this arm will receive volixibat 20mg twice daily. Receives: Drug: Volixibat |
| Part 1 Arm 2: Volixibat 80mg | Experimental | Participants randomized to this arm will receive volixibat 80mg twice daily. Receives: Drug: Volixibat |
| Part 1 Arm 3: Placebo | Placebo comparator | Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily. Receives: Drug: Placebo |
| Part 2 Arm 1: Volixibat Selected Dose 20mg | Experimental | Participants randomized to this arm will receive volixibat 20mg twice daily. Receives: Drug: Volixibat |
| Part 2 Arm 2: Placebo | Placebo comparator | Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline to week 28
Secondary outcomes (8)
Proportion of participants with itch response using the Adult ItchRO
Measured: Baseline to week 28
Incidence of adverse events
Measured: Baseline to week 28
Changes in alkaline phosphatase
Measured: Baseline to week 28
Changes in total bilirubin
Measured: Baseline to week 28
Changes in serum bile acid levels
Measured: Baseline to week 28
Change in Primary Biliary Cholangitis Quality of Life Measure (PBC-40)
PBC-40 questionnaire is scored on a scale of 40 to 200, with higher scores indicating poorer quality of life
Measured: Baseline to week 28
Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) fatigue questionnaire
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.
Measured: Baseline to week 28
Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) sleep disturbance questionnaire
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.
Measured: Baseline to week 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Sep 2021
- Study start22 Sep 2021
- Primary completionJan 2027 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Primary Biliary CholangitisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 333 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05050136
- Sponsor's protocol ID
- VLX-601
- Acronym
- VANTAGE
- Status last verified
- Aug 2026
- First submitted
- 9 Sep 2021
- Last updated
- 25 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
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.
PubMed 40439725