A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC)
NCT ID: NCT04663308
Last Updated: 2026-01-15
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
ACTIVE_NOT_RECRUITING
PHASE2
182 participants
INTERVENTIONAL
2020-12-18
2027-04-30
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Biliary Cholangitis
NCT05050136
Volixibat (SHP626) in the Treatment of Adults With Nonalcoholic Steatohepatitis (NASH)
NCT02787304
Safety and Tolerability of A3907 in Primary Sclerosing Cholangitis
NCT05642468
Study with Norucholic Acid Tablets in Patients with Primary Sclerosing Cholangitis (PSC)
NCT06886360
Study to Evaluate EP547 in Subjects With Cholestatic Pruritus Due to Primary Biliary Cholangitis or Primary Sclerosing Cholangitis
NCT05525520
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Part 1 Arm 1: Volixibat 20mg
Participants randomized to this arm will receive volixibat 20mg twice daily.
Volixibat
Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.
Part 1 Arm 2: Volixibat 80mg
Participants randomized to this arm will receive volixibat 80mg twice daily.
Volixibat
Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.
Part 1 Arm 3: Placebo
Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
Placebo
Capsules matched to study drug minus active substance
Part 2 Arm 1: Volixibat Selected Dose 20mg
Participants randomized to this arm will receive volixibat 20mg twice daily.
Volixibat
Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.
Part 2 Arm 2: Placebo
Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
Placebo
Capsules matched to study drug minus active substance
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Volixibat
Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.
Placebo
Capsules matched to study drug minus active substance
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
2. Subjects aged ≥12 years for eligible regions; otherwise ≥18 years
3. Confirmed diagnosis of large duct or small duct PSC based on American Association for the Study of Liver Disease (AASLD) guidelines.
4. Pruritus associated with PSC as assessed by Adult ItchRO.
5. Ursodeoxycholic acid (UDCA) and anti-pruritic medication use will be allowed if meeting additional criteria.
6. Concomitant Inflammatory Bowel Disease (IBD) is allowed if meeting additional criteria.
Exclusion Criteria
2. Evidence or clinical suspicion of decompensated cirrhosis, or a history of decompensation events
3. History of ileostomy or small bowel surgery/resection or other surgeries that may have disrupted the enterohepatic circulation
4. Evidence, history, or suspicion of other liver disease; PSC patients with AIH are not excluded.
5. Bile duct stent or percutaneous bile duct drain placement, or balloon dilatation procedure of a stricture within 12 weeks of Screening
6. 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
7. History of liver transplantation
12 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Mirum Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Southern California Research Center
Coronado, California, United States
Cedars Sinai Medical Center
Los Angeles, California, United States
University of California, Davis
Sacramento, California, United States
California Pacific Medical Center Research Institute
San Francisco, California, United States
University of Colorado - Anschutz Medical Campus
Aurora, Colorado, United States
MedStar Georgetown University Hospital
Washington D.C., District of Columbia, United States
Florida Research Institute
Lakewood Rch, Florida, United States
University of Miami - Schiff Center for Liver Diseases
Miami, Florida, United States
Tampa General Hospital
Tampa, Florida, United States
Beth Israel Deaconess Medical Center
Boston, Massachusetts, United States
Henry Ford Hospital
Detroit, Michigan, United States
Mayo Clinic
Rochester, Minnesota, United States
Southern Therapy and Advanced Research LLC
Jackson, Mississippi, United States
Northwell Health
Manhasset, New York, United States
New York University Langone Health
New York, New York, United States
Weill Cornell Medical College
New York, New York, United States
Columbia University Medical Center - Presbyterian Hospital and Vanderbilt Clinic
New York, New York, United States
University of Rochester Medical Center - Strong Memorial Hospital
Rochester, New York, United States
Duke Health - Duke University Medical Center
Durham, North Carolina, United States
Science 37, Inc (Remote-homebased Telemedicine)
Morrisville, North Carolina, United States
Cleveland Clinic
Cleveland, Ohio, United States
Einstein Healthcare Network - Einstein Medical Center
Philadelphia, Pennsylvania, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States
Galen Medical Group
Hixson, Tennessee, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
The Liver Institute At Methodist Dallas Medical Center
Dallas, Texas, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
Houston Methodist Hospital
Houston, Texas, United States
Liver Associates of Texas
Houston, Texas, United States
University of Utah Health Care
Salt Lake City, Utah, United States
Bon Secours Liver Institution of Hampton Roads Mary Immaculate Hospital Office
Newport News, Virginia, United States
Richmond Community Hospital LLC
Richmond, Virginia, United States
University of Washington - Harborview Medical Center
Seattle, Washington, United States
Liver Institute Northwest
Seattle, Washington, United States
Hospital Italiano de Buenos Aires
Buenos Aires, , Argentina
Glenny Corp S.A.
Ciudad Autónoma Buenos Aires, , Argentina
Hospital El Cruce
San Juan Bautista, , Argentina
Royal Adelaide Hospital
Adelaide, , Australia
Northern Health
Epping, , Australia
Liverpool Hospital
Liverpool, , Australia
Austin Health
Melbourne, , Australia
University of the Sunshine Coast
Sippy Downs, , Australia
Cliniques Universitaires Saint-Luc
Brussels, , Belgium
Universitair Ziekenhuis Antwerpen (UZA)
Ghent, , Belgium
University Hospital Ghent
Ghent, , Belgium
Hospital das Clinicas da UFMG
Belo Horizonte, , Brazil
Hospital Das Clínicas Da Ufg
Goiânia, , Brazil
Hospital de Clinicas de Porto Alegre
Porto Alegre, , Brazil
Escola Bahiana de Medicina e Saude Publica
Salvador, , Brazil
Hospital do Rim e Hipertensao
São Paulo, , Brazil
University of Alberta
Edmonton, Alberta, Canada
McMaster University Hospital
Hamilton, Ontario, Canada
The Ottawa Hospital - General Campus
Ottawa, Ontario, Canada
University Health Network - Toronto General Hospital
Toronto, Ontario, Canada
University of Calgary
Calgary, , Canada
London Health Sciences Centre University Hospital
London, , Canada
McGill University Health Centre - Royal Victoria Hospital
Montreal, , Canada
Centre Hospitalier Universitaire Grenoble Alpes (CHU Grenoble Alpes)
Grenoble, , France
Centre Hospitalier Universitaire de Lille
Lille, , France
APHP-Hôpital Saint Antoine
Paris, , France
CHU Bordeaux - Haut Lévêque
Pessac, , France
Hannover Medical School
Hanover, Lower Saxony, Germany
Universitaetsklinikum Bonn
Bonn, Nordrhine-Westphalia, Germany
Charité Universtitätsmedizin - Campus Virchow-Klinikum
Berlin, , Germany
Universitaetsklinikum Erlangen
Erlangen, , Germany
Universitätsklinikum Schleswig-Holstein - Campus Kiel
Kiel, , Germany
Universitatsklinikum Leipzig
Leipzig, , Germany
Otto-von-Guericke-Universitat Magdeburg
Magdeburg, , Germany
University Hospital Tubingen Medical Clinic
Tübingen, , Germany
St. Josefs-Hospital Wiesbaden
Wiesbaden, , Germany
Hillel Yaffe Medical Center
Hadera, , Israel
Rambam Medical Center
Haifa, , Israel
Carmel Medical Center
Haifa, , Israel
Shaare Zedek Medical Center
Jerusalem, , Israel
Hadassah Medical Center - Ein Karem
Jerusalem, , Israel
Galilee Medical Center
Nahariya, , Israel
Rabin Medical Center
Petah Tikva, , Israel
Sheba Medical Center
Ramat Gan, , Israel
Tel Aviv Sourasky Medical Center
Tel Aviv, , Israel
Azienda Ospedaliero-Universitaria di Bologna - Policlinico S. Orsola-Malpighi
Bologna, , Italy
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Milan, , Italy
ASST Grande Ospedale Metropolitano Niguarda
Milan, , Italy
Fondazione IRCCS San Gerardo dei Tintori
Monza, , Italy
A.O.U. Federico II
Napoli, , Italy
Azienda Ospedale Universita Padova
Padua, , Italy
Amsterdam UMC - VU Medisch Centrum (VUmc)
Amsterdam, , Netherlands
Universitair Medisch Centrum Groningen
Groningen, , Netherlands
Radboud University Medical Centre (MC)
Nijmegen, , Netherlands
Erasmus University Medical Center
Rotterdam, , Netherlands
Hospital Clinic Barcelona
Barcelona, , Spain
Hospital General Universitario Gregorio Maranon
Madrid, , Spain
University Hospital Zurich
Zurich, , Switzerland
Royal Free Hospital
London, Hampstead, United Kingdom
John Radcliffe Hospital
Oxford, Headington, United Kingdom
Royal Victoria Hospital
Belfast, , United Kingdom
University Hospitals Birmingham NHS Foundation Trust - Wellcome Trust Birmingham Clinical Research Facility
Birmingham, , United Kingdom
Cambridge University Hospitals NHS Foundation Trust - Addenbrooke's Hospital
Cambridge, , United Kingdom
Lothian NHS Board
Edinburgh, , United Kingdom
St James' University Hospital Leeds Teaching Hospitals NHS Trust
Leeds, , United Kingdom
King's College Hospital NHS Foundation Trust
London, , United Kingdom
The Newcastle upon Tyne Hospitals NHS Foundation Trust - Royal Victoria Infirmary
Newcastle upon Tyne, , United Kingdom
Nottingham University Hospital NHS Trust
Nottingham, , United Kingdom
University Hospital Southampton NHS Foundation Trust
Southampton, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
2020-003027-41
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
2023-505764-11-00
Identifier Type: OTHER
Identifier Source: secondary_id
VLX-301
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.