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New hope for PSC patients: drug targets debilitating itch

NCT ID NCT04663308

Symptom relief Sponsor: Mirum Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 27, 2026 · Updated 2 times

Summary

This study tests a medicine called volixibat to see if it can reduce severe itching in people with primary sclerosing cholangitis (PSC), a rare liver disease. About 182 participants will receive either volixibat or a placebo, and researchers will track itch scores using a daily diary. The goal is to find a safe treatment that improves quality of life and may also slow disease progression.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

182 people

The number who actually took part.

Started

Dec 2020

Expected to finish

Mar 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. 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. 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: 1. Pruritus associated with an etiology other than PSC 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

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Conditions

The condition(s) this trial relates to.

Cholangitis, Sclerosing cholestasis primary sclerosing cholangitis Pruritus

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Primary sclerosing cholangitis

Contacts and locations

Locations

  • A.O.U. Federico II

    Naples, 80131, Italy

  • APHP-Hôpital Saint Antoine

    Paris, 75571, France

  • ASST Grande Ospedale Metropolitano Niguarda

    Milan, 20162, Italy

  • Amsterdam UMC - VU Medisch Centrum (VUmc)

    Amsterdam, 1081 HV, Netherlands

  • Austin Health

    Melbourne, 3084, Australia

  • Azienda Ospedale Universita Padova

    Padova, 35100, Italy

  • Azienda Ospedaliero-Universitaria di Bologna - Policlinico S. Orsola-Malpighi

    Bologna, 40138, Italy

  • Beth Israel Deaconess Medical Center

    Boston, Massachusetts, 02215, United States

  • Bon Secours Liver Institution of Hampton Roads Mary Immaculate Hospital Office

    Newport News, Virginia, 23603, United States

  • CHU Bordeaux - Haut Lévêque

    Pessac, 33600, France

  • California Pacific Medical Center Research Institute

    San Francisco, California, 94109, United States

  • Cambridge University Hospitals NHS Foundation Trust - Addenbrooke's Hospital

    Cambridge, CB2 0QQ, United Kingdom

  • Carmel Medical Center

    Haifa, 3434104, Israel

  • Cedars Sinai Medical Center

    Los Angeles, California, 90048, United States

  • Centre Hospitalier Universitaire De Lille

    Lille, 59037, France

  • Centre Hospitalier Universitaire Grenoble Alpes (CHU Grenoble Alpes)

    Grenoble, 38043, France

  • Charité Universtitätsmedizin - Campus Virchow-Klinikum

    Berlin, 13353, Germany

  • Cleveland Clinic

    Cleveland, Ohio, 44195, United States

  • Cliniques Universitaires Saint-Luc

    Brussels, 1200, Belgium

  • Columbia University Medical Center - Presbyterian Hospital and Vanderbilt Clinic

    New York, New York, 10032, United States

  • Duke Health - Duke University Medical Center

    Durham, North Carolina, 27710, United States

  • Einstein Healthcare Network - Einstein Medical Center

    Philadelphia, Pennsylvania, 19141, United States

  • Erasmus University Medical Center

    Rotterdam, 3015 GD, Netherlands

  • Escola Bahiana de Medicina e Saude Publica

    Salvador, 40290-000, Brazil

  • Florida Research Institute

    Lakewood Rch, Florida, 34211, United States

  • Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico

    Milan, 20122, Italy

  • Fondazione IRCCS San Gerardo dei Tintori

    Monza, 20900, Italy

  • Galen Medical Group

    Hixson, Tennessee, 37343, United States

  • Galilee Medical Center

    Nahariya, 2210001, Israel

  • Glenny Corp S.A.

    Ciudad Autónoma Buenos Aires, C1430, Argentina

  • Hadassah Medical Center - Ein Karem

    Jerusalem, 91120, Israel

  • Hannover Medical School

    Hanover, Lower Saxony, 30625, Germany

  • Henry Ford Hospital

    Detroit, Michigan, 48202, United States

  • Hillel Yaffe Medical Center

    Hadera, 38100, Israel

  • Hospital Clinic Barcelona

    Barcelona, 08036, Spain

  • Hospital Das Clinicas Da Ufg

    Goiânia, 74535-170, Brazil

  • Hospital El Cruce

    San Juan Bautista, B1888, Argentina

  • Hospital General Universitario Gregorio Maranon

    Madrid, 28007, Spain

  • Hospital Italiano de Buenos Aires

    Buenos Aires, C1199, Argentina

  • Hospital das Clinicas da UFMG

    Belo Horizonte, 30140-080, Brazil

  • Hospital de Clinicas de Porto Alegre

    Porto Alegre, 90035-903, Brazil

  • Hospital do Rim e Hipertensao

    São Paulo, 04038-002, Brazil

  • Houston Methodist Hospital

    Houston, Texas, 77030, United States

  • John Radcliffe Hospital

    Oxford, Headington, OX3 9DU, United Kingdom

  • King's College Hospital NHS Foundation Trust

    London, SE5 9RS, United Kingdom

  • Liver Associates of Texas

    Houston, Texas, 77030, United States

  • Liver Institute Northwest

    Seattle, Washington, 98105, United States

  • Liverpool Hospital

    Liverpool, 2170, Australia

  • London Health Sciences Centre University Hospital

    London, N6A 5A5, Canada

  • Lothian NHS Board

    Edinburgh, EH164SA, United Kingdom

  • Mayo Clinic

    Rochester, Minnesota, 55905, United States

  • McGill University Health Centre - Royal Victoria Hospital

    Montreal, H4A 3J1, Canada

  • McMaster University Hospital

    Hamilton, Ontario, L8L 0A4, Canada

  • MedStar Georgetown University Hospital

    Washington D.C., District of Columbia, 20007, United States

  • New York University Langone Health

    New York, New York, 10016, United States

  • Northern Health

    Epping, VIC 3076, Australia

  • Northwell Health

    Manhasset, New York, 11030, United States

  • Nottingham University Hospital NHS Trust

    Nottingham, NG7 2FT, United Kingdom

  • Otto-von-Guericke-Universitat Magdeburg

    Magdeburg, 39120, Germany

  • Rabin Medical Center

    Petah Tikva, 4941492, Israel

  • Radboud University Medical Centre (MC)

    Nijmegen, 6525 GA, Netherlands

  • Rambam Medical Center

    Haifa, 3109601, Israel

  • Richmond Community Hospital LLC

    Richmond, Virginia, 23226, United States

  • Royal Adelaide Hospital

    Adelaide, 5000, Australia

  • Royal Free Hospital

    London, Hampstead, NW3 2QG, United Kingdom

  • Royal Victoria Hospital

    Belfast, BT12 6BA, United Kingdom

  • Science 37, Inc (Remote-homebased Telemedicine)

    Morrisville, North Carolina, 27560, United States

  • Shaare Zedek Medical Center

    Jerusalem, 9103102, Israel

  • Sheba Medical Center

    Ramat Gan, 52621, Israel

  • Southern California Research Center

    Coronado, California, 92118, United States

  • Southern Therapy and Advanced Research LLC

    Jackson, Mississippi, 39216, United States

  • St James' University Hospital Leeds Teaching Hospitals NHS Trust

    Leeds, LS9 7TF, United Kingdom

  • St. Josefs-Hospital Wiesbaden

    Wiesbaden, 65189, Germany

  • Tampa General Hospital

    Tampa, Florida, 33606, United States

  • Tel Aviv Sourasky Medical Center

    Tel Aviv, 6423906, Israel

  • The Liver Institute At Methodist Dallas Medical Center

    Dallas, Texas, 75203, United States

  • The Newcastle upon Tyne Hospitals NHS Foundation Trust - Royal Victoria Infirmary

    Newcastle upon Tyne, NE1 4LP, United Kingdom

  • The Ottawa Hospital - General Campus

    Ottawa, Ontario, Canada

  • Universitaetsklinikum Bonn

    Bonn, Nordrhine-Westphalia, 53127, Germany

  • Universitaetsklinikum Erlangen

    Erlangen, 91054, Germany

  • Universitair Medisch Centrum Groningen

    Groningen, 9713 GZ, Netherlands

  • Universitair Ziekenhuis Antwerpen (UZA)

    Ghent, 9000, Belgium

  • Universitatsklinikum Leipzig

    Leipzig, 04103, Germany

  • University Health Network - Toronto General Hospital

    Toronto, Ontario, M5G 2C4, Canada

  • University Hospital Ghent

    Ghent, 9000, Belgium

  • University Hospital Southampton NHS Foundation Trust

    Southampton, United Kingdom

  • University Hospital Tubingen Medical Clinic

    Tübingen, 72076, Germany

  • University Hospital Zurich

    Zurich, 8091, Switzerland

  • University Hospitals Birmingham NHS Foundation Trust - Wellcome Trust Birmingham Clinical Research Facility

    Birmingham, B15 2TH, United Kingdom

  • University of Alberta

    Edmonton, Alberta, T6G 2R3, Canada

  • University of Calgary

    Calgary, T2N 4Z6, Canada

  • University of California, Davis

    Sacramento, California, 95817, United States

  • University of Colorado - Anschutz Medical Campus

    Aurora, Colorado, 80045, United States

  • University of Miami - Schiff Center for Liver Diseases

    Miami, Florida, 33136, United States

  • University of Pittsburgh Medical Center

    Pittsburgh, Pennsylvania, 15213, United States

  • University of Rochester Medical Center - Strong Memorial Hospital

    Rochester, New York, 14642, United States

  • University of Texas Southwestern Medical Center

    Dallas, Texas, 75390, United States

  • University of Utah Health Care

    Salt Lake City, Utah, 84132, United States

  • University of Washington - Harborview Medical Center

    Seattle, Washington, 98104, United States

  • University of the Sunshine Coast

    Sippy Downs, 4556, Australia

  • Universitätsklinikum Schleswig-Holstein - Campus Kiel

    Kiel, 24105, Germany

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232, United States

  • Weill Cornell Medical College

    New York, New York, 10021, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.