A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC)
VISTAS
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 Sclerosing Cholangitis
3 other identifiers
interventional
182
14 countries
103
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Dec 2020
Longer than P75 for phase_2
103 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
November 17, 2020
CompletedFirst Posted
Study publicly available on registry
December 11, 2020
CompletedStudy Start
First participant enrolled
December 18, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 1, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
April 1, 2027
February 4, 2026
February 1, 2026
5.5 years
November 17, 2020
February 2, 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 through to Week 28
Secondary Outcomes (8)
Proportion of participants with itch response using the Adult ItchRO
Baseline through to Week 28
The incidence of adverse events
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
- +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 minus active substance
Eligibility Criteria
You may qualify if:
- 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.
- Subjects aged ≥12 years for eligible regions; otherwise ≥18 years
- Confirmed diagnosis of large duct or small duct PSC based on American Association for the Study of Liver Disease (AASLD) guidelines.
- Pruritus associated with PSC as assessed by Adult ItchRO.
- Ursodeoxycholic acid (UDCA) and anti-pruritic medication use will be allowed if meeting additional criteria.
- Concomitant Inflammatory Bowel Disease (IBD) is allowed if meeting additional criteria.
You may not qualify if:
- Pruritus associated with an etiology other than PSC
- Evidence or clinical suspicion of decompensated cirrhosis, or a history of decompensation events
- History of ileostomy or small bowel surgery/resection or other surgeries that may have disrupted the enterohepatic circulation
- Evidence, history, or suspicion of other liver disease; PSC patients with AIH are not excluded.
- Bile duct stent or percutaneous bile duct drain placement, or balloon dilatation procedure of a stricture within 12 weeks of Screening
- 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
- History of liver transplantation
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (103)
Southern California Research Center
Coronado, California, 92118, United States
Cedars Sinai Medical Center
Los Angeles, California, 90048, United States
University of California, Davis
Sacramento, California, 95817, United States
California Pacific Medical Center Research Institute
San Francisco, California, 94109, United States
University of Colorado - Anschutz Medical Campus
Aurora, Colorado, 80045, United States
MedStar Georgetown University Hospital
Washington D.C., District of Columbia, 20007, United States
Florida Research Institute
Lakewood Rch, Florida, 34211, United States
University of Miami - Schiff Center for Liver Diseases
Miami, Florida, 33136, United States
Tampa General Hospital
Tampa, Florida, 33606, United States
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215, United States
Henry Ford Hospital
Detroit, Michigan, 48202, United States
Mayo Clinic
Rochester, Minnesota, 55905, United States
Southern Therapy and Advanced Research LLC
Jackson, Mississippi, 39216, United States
Northwell Health
Manhasset, New York, 11030, United States
New York University Langone Health
New York, New York, 10016, United States
Weill Cornell Medical College
New York, New York, 10021, United States
Columbia University Medical Center - Presbyterian Hospital and Vanderbilt Clinic
New York, New York, 10032, United States
University of Rochester Medical Center - Strong Memorial Hospital
Rochester, New York, 14642, United States
Duke Health - Duke University Medical Center
Durham, North Carolina, 27710, United States
Science 37, Inc (Remote-homebased Telemedicine)
Morrisville, North Carolina, 27560, United States
Cleveland Clinic
Cleveland, Ohio, 44195, United States
Einstein Healthcare Network - Einstein Medical Center
Philadelphia, Pennsylvania, 19141, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213, United States
Galen Medical Group
Hixson, Tennessee, 37343, United States
Vanderbilt University Medical Center
Nashville, Tennessee, 37232, United States
The Liver Institute At Methodist Dallas Medical Center
Dallas, Texas, 75203, United States
University of Texas Southwestern Medical Center
Dallas, Texas, 75390, United States
Houston Methodist Hospital
Houston, Texas, 77030, United States
Liver Associates of Texas
Houston, Texas, 77030, United States
University of Utah Health Care
Salt Lake City, Utah, 84132, United States
Bon Secours Liver Institution of Hampton Roads Mary Immaculate Hospital Office
Newport News, Virginia, 23603, United States
Richmond Community Hospital LLC
Richmond, Virginia, 23226, United States
University of Washington - Harborview Medical Center
Seattle, Washington, 98104, United States
Liver Institute Northwest
Seattle, Washington, 98105, United States
Hospital Italiano de Buenos Aires
Buenos Aires, C1199, Argentina
Glenny Corp S.A.
Ciudad AutĂ³noma Buenos Aires, C1430, Argentina
Hospital El Cruce
San Juan Bautista, B1888, Argentina
Royal Adelaide Hospital
Adelaide, 5000, Australia
Northern Health
Epping, VIC 3076, Australia
Liverpool Hospital
Liverpool, 2170, Australia
Austin Health
Melbourne, 3084, Australia
University of the Sunshine Coast
Sippy Downs, 4556, Australia
Cliniques Universitaires Saint-Luc
Brussels, 1200, Belgium
Universitair Ziekenhuis Antwerpen (UZA)
Ghent, 9000, Belgium
University Hospital Ghent
Ghent, 9000, Belgium
Hospital das Clinicas da UFMG
Belo Horizonte, 30140-080, Brazil
Hospital Das Clinicas Da Ufg
GoiĂ¢nia, 74535-170, Brazil
Hospital de Clinicas de Porto Alegre
Porto Alegre, 90035-903, Brazil
Escola Bahiana de Medicina e Saude Publica
Salvador, 40290-000, Brazil
Hospital do Rim e Hipertensao
SĂ£o Paulo, 04038-002, Brazil
University of Alberta
Edmonton, Alberta, T6G 2R3, Canada
McMaster University Hospital
Hamilton, Ontario, L8L 0A4, Canada
The Ottawa Hospital - General Campus
Ottawa, Ontario, Canada
University Health Network - Toronto General Hospital
Toronto, Ontario, M5G 2C4, Canada
University of Calgary
Calgary, T2N 4Z6, Canada
London Health Sciences Centre University Hospital
London, N6A 5A5, Canada
McGill University Health Centre - Royal Victoria Hospital
Montreal, H4A 3J1, Canada
Centre Hospitalier Universitaire Grenoble Alpes (CHU Grenoble Alpes)
Grenoble, 38043, France
Centre Hospitalier Universitaire De Lille
Lille, 59037, France
APHP-HĂ´pital Saint Antoine
Paris, 75571, France
CHU Bordeaux - Haut LĂ©vĂªque
Pessac, 33600, France
Hannover Medical School
Hanover, Lower Saxony, 30625, Germany
Universitaetsklinikum Bonn
Bonn, Nordrhine-Westphalia, 53127, Germany
Charité Universtitätsmedizin - Campus Virchow-Klinikum
Berlin, 13353, Germany
Universitaetsklinikum Erlangen
Erlangen, 91054, Germany
Universitätsklinikum Schleswig-Holstein - Campus Kiel
Kiel, 24105, Germany
Universitatsklinikum Leipzig
Leipzig, 04103, Germany
Otto-von-Guericke-Universitat Magdeburg
Magdeburg, 39120, Germany
University Hospital Tubingen Medical Clinic
TĂ¼bingen, 72076, Germany
St. Josefs-Hospital Wiesbaden
Wiesbaden, 65189, Germany
Hillel Yaffe Medical Center
Hadera, 38100, 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
Jerusalem, 91120, Israel
Galilee Medical Center
Nahariya, 2210001, Israel
Rabin Medical Center
Petah Tikva, 4941492, Israel
Sheba Medical Center
Ramat Gan, 52621, Israel
Tel Aviv Sourasky Medical Center
Tel Aviv, 6423906, Israel
Azienda Ospedaliero-Universitaria di Bologna - Policlinico S. Orsola-Malpighi
Bologna, 40138, Italy
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Milan, 20122, Italy
ASST Grande Ospedale Metropolitano Niguarda
Milan, 20162, Italy
Fondazione IRCCS San Gerardo dei Tintori
Monza, 20900, Italy
A.O.U. Federico II
Naples, 80131, Italy
Azienda Ospedale Universita Padova
Padua, 35100, Italy
Amsterdam UMC - VU Medisch Centrum (VUmc)
Amsterdam, 1081 HV, Netherlands
Universitair Medisch Centrum Groningen
Groningen, 9713 GZ, Netherlands
Radboud University Medical Centre (MC)
Nijmegen, 6525 GA, Netherlands
Erasmus University Medical Center
Rotterdam, 3015 GD, Netherlands
Hospital Clinic Barcelona
Barcelona, 08036, Spain
Hospital General Universitario Gregorio Maranon
Madrid, 28007, Spain
University Hospital Zurich
Zurich, 8091, Switzerland
Royal Free Hospital
London, Hampstead, NW3 2QG, United Kingdom
John Radcliffe Hospital
Oxford, Headington, OX3 9DU, United Kingdom
Royal Victoria Hospital
Belfast, BT12 6BA, United Kingdom
University Hospitals Birmingham NHS Foundation Trust - Wellcome Trust Birmingham Clinical Research Facility
Birmingham, B15 2TH, United Kingdom
Cambridge University Hospitals NHS Foundation Trust - Addenbrooke's Hospital
Cambridge, CB2 0QQ, United Kingdom
Lothian NHS Board
Edinburgh, EH164SA, United Kingdom
St James' University Hospital Leeds Teaching Hospitals NHS Trust
Leeds, LS9 7TF, United Kingdom
King's College Hospital NHS Foundation Trust
London, SE5 9RS, United Kingdom
The Newcastle upon Tyne Hospitals NHS Foundation Trust - Royal Victoria Infirmary
Newcastle upon Tyne, NE1 4LP, United Kingdom
Nottingham University Hospital NHS Trust
Nottingham, NG7 2FT, United Kingdom
University Hospital Southampton NHS Foundation Trust
Southampton, United Kingdom
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
November 17, 2020
First Posted
December 11, 2020
Study Start
December 18, 2020
Primary Completion (Estimated)
June 1, 2026
Study Completion (Estimated)
April 1, 2027
Last Updated
February 4, 2026
Record last verified: 2026-02