Primary Sclerosing CholangitisPhase 2VISTASNCT04663308
A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC)
A Phase 2 study testing Volixibat in people with Primary Sclerosing Cholangitis, run by Mirum Pharmaceuticals, Inc. at 103 sites in 14 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 Sclerosing Cholangitis (PSC), and to assess the possible impact on the disease progression of PSC. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Primary Sclerosing Cholangitis
- 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
- 182 participants
- Where
- 103 sitesin 14 countries
- Study dates
- 18 Dec 2020to Mar 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
103 sites in 14 countries.
103 sites in 14 countries
United States34 sites
University of Colorado - Anschutz Medical Campus
Aurora, Colorado, 80045
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
Cleveland Clinic
Cleveland, Ohio, 44195
Southern California Research Center
Coronado, California, 92118
The Liver Institute At Methodist Dallas Medical Center
Dallas, Texas, 75203
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
Henry Ford Hospital
Detroit, Michigan, 48202
Duke Health - Duke University Medical Center
Durham, North Carolina, 27710
Galen Medical Group
Hixson, Tennessee, 37343
Houston Methodist Hospital
Houston, Texas, 77030
Liver Associates of Texas
Houston, Texas, 77030
Southern Therapy and Advanced Research LLC
Jackson, Mississippi, 39216
Florida Research Institute
Lakewood Rch, Florida, 34211
Cedars Sinai Medical Center
Los Angeles, California, 90048
Northwell Health
Manhasset, New York, 11030
University of Miami - Schiff Center for Liver Diseases
Miami, Florida, 33136
Science 37, Inc (Remote-homebased Telemedicine)
Morrisville, North Carolina, 27560
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
New York University Langone Health
New York, New York, 10016
Weill Cornell Medical College
New York, New York, 10021
Columbia University Medical Center - Presbyterian Hospital and Vanderbilt Clinic
New York, New York, 10032
Bon Secours Liver Institution of Hampton Roads Mary Immaculate Hospital Office
Newport News, Virginia, 23603
Einstein Healthcare Network - Einstein Medical Center
Philadelphia, Pennsylvania, 19141
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213
Richmond Community Hospital LLC
Richmond, Virginia, 23226
Mayo Clinic
Rochester, Minnesota, 55905
University of Rochester Medical Center - Strong Memorial Hospital
Rochester, New York, 14642
University of California, Davis
Sacramento, California, 95817
University of Utah Health Care
Salt Lake City, Utah, 84132
California Pacific Medical Center Research Institute
San Francisco, California, 94109
University of Washington - Harborview Medical Center
Seattle, Washington, 98104
Liver Institute Northwest
Seattle, Washington, 98105
Tampa General Hospital
Tampa, Florida, 33606
MedStar Georgetown University Hospital
Washington D.C., District of Columbia, 20007
United Kingdom11 sites
Royal Victoria Hospital
Belfast, BT12 6BA
University Hospitals Birmingham NHS Foundation Trust - Wellcome Trust Birmingham Clinical Research Facility
Birmingham, B15 2TH
Cambridge University Hospitals NHS Foundation Trust - Addenbrooke's Hospital
Cambridge, CB2 0QQ
Lothian NHS Board
Edinburgh, EH164SA
St James' University Hospital Leeds Teaching Hospitals NHS Trust
Leeds, LS9 7TF
Royal Free Hospital
London, Hampstead, NW3 2QG
King's College Hospital NHS Foundation Trust
London, SE5 9RS
The Newcastle upon Tyne Hospitals NHS Foundation Trust - Royal Victoria Infirmary
Newcastle upon Tyne, NE1 4LP
Nottingham University Hospital NHS Trust
Nottingham, NG7 2FT
John Radcliffe Hospital
Oxford, Headington, OX3 9DU
University Hospital Southampton NHS Foundation Trust
Southampton
Germany9 sites
Charité Universtitätsmedizin - Campus Virchow-Klinikum
Berlin, 13353
Universitaetsklinikum Bonn
Bonn, Nordrhine-Westphalia, 53127
Universitaetsklinikum Erlangen
Erlangen, 91054
Hannover Medical School
Hanover, Lower Saxony, 30625
Universitätsklinikum Schleswig-Holstein - Campus Kiel
Kiel, 24105
Universitatsklinikum Leipzig
Leipzig, 04103
Otto-von-Guericke-Universitat Magdeburg
Magdeburg, 39120
University Hospital Tubingen Medical Clinic
Tübingen, 72076
St. Josefs-Hospital Wiesbaden
Wiesbaden, 65189
Israel9 sites
Hillel Yaffe Medical Center
Hadera, 38100
Rambam Medical Center
Haifa, 3109601
Carmel Medical Center
Haifa, 3434104
Shaare Zedek Medical Center
Jerusalem, 9103102
Hadassah Medical Center - Ein Karem
Jerusalem, 91120
Galilee Medical Center
Nahariya, 2210001
Rabin Medical Center
Petah Tikva, 4941492
Sheba Medical Center
Ramat Gan, 52621
Tel Aviv Sourasky Medical Center
Tel Aviv, 6423906
Canada7 sites
University of Calgary
Calgary, T2N 4Z6
University of Alberta
Edmonton, Alberta, T6G 2R3
McMaster University Hospital
Hamilton, Ontario, L8L 0A4
London Health Sciences Centre University Hospital
London, N6A 5A5
McGill University Health Centre - Royal Victoria Hospital
Montreal, H4A 3J1
The Ottawa Hospital - General Campus
Ottawa, Ontario
University Health Network - Toronto General Hospital
Toronto, Ontario, M5G 2C4
Italy6 sites
Azienda Ospedaliero-Universitaria di Bologna - Policlinico S. Orsola-Malpighi
Bologna, 40138
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Milan, 20122
ASST Grande Ospedale Metropolitano Niguarda
Milan, 20162
Fondazione IRCCS San Gerardo dei Tintori
Monza, 20900
A.O.U. Federico II
Naples, 80131
Azienda Ospedale Universita Padova
Padova, 35100
Australia5 sites
Royal Adelaide Hospital
Adelaide, 5000
Northern Health
Epping, VIC 3076
Liverpool Hospital
Liverpool, 2170
Austin Health
Melbourne, 3084
University of the Sunshine Coast
Sippy Downs, 4556
Brazil5 sites
Hospital das Clinicas da UFMG
Belo Horizonte, 30140-080
Hospital Das Clinicas Da Ufg
Goiânia, 74535-170
Hospital de Clinicas de Porto Alegre
Porto Alegre, 90035-903
Escola Bahiana de Medicina e Saude Publica
Salvador, 40290-000
Hospital do Rim e Hipertensao
São Paulo, 04038-002
France4 sites
Centre Hospitalier Universitaire Grenoble Alpes (CHU Grenoble Alpes)
Grenoble, 38043
Centre Hospitalier Universitaire De Lille
Lille, 59037
APHP-Hôpital Saint Antoine
Paris, 75571
CHU Bordeaux - Haut Lévêque
Pessac, 33600
Netherlands4 sites
Amsterdam UMC - VU Medisch Centrum (VUmc)
Amsterdam, 1081 HV
Universitair Medisch Centrum Groningen
Groningen, 9713 GZ
Radboud University Medical Centre (MC)
Nijmegen, 6525 GA
Erasmus University Medical Center
Rotterdam, 3015 GD
Argentina3 sites
Hospital Italiano de Buenos Aires
Buenos Aires, C1199
Glenny Corp S.A.
Ciudad Autónoma Buenos Aires, C1430
Hospital El Cruce
San Juan Bautista, B1888
Belgium3 sites
Cliniques Universitaires Saint-Luc
Brussels, 1200
Universitair Ziekenhuis Antwerpen (UZA)
Ghent, 9000
University Hospital Ghent
Ghent, 9000
Spain2 sites
Hospital Clinic Barcelona
Barcelona, 08036
Hospital General Universitario Gregorio Maranon
Madrid, 28007
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 Sclerosing 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 Sclerosing Cholangitis (PSC), and to assess the possible impact on the disease progression of PSC.
- Conditions
- Primary Sclerosing Cholangitis
- Being tested
- Volixibat, Placebo
- Keywords
- Pruritus, PSC, Itch, Itching, Cholestasis
- Medical subject headings
- Cholangitis, Sclerosing, Pruritus, Cholestasis
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 minus active substance
| 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 through to Week 28
Secondary outcomes (8)
Proportion of participants with itch response using the Adult ItchRO
Measured: Baseline through to Week 28
The incidence of adverse events
Measured: Baseline through to Week 28
Changes in serum bile acid levels
Measured: Baseline through to Week 28
Changes in alkaline phosphatase
Measured: Baseline through to Week 28
Changes in total bilirubin levels
Measured: Baseline through to Week 28
Change in Primary Sclerosing Cholangitis-Specific Patient-Reported Outcome
Measured: Baseline through 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 through 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 through 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 posted11 Dec 2020
- Study start18 Dec 2020
- Primary completion20 Feb 2026
- Study completionMar 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Sclerosing 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
- 182 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
- NCT04663308
- Sponsor's protocol ID
- VLX-301
- Other IDs
- 2020-003027-41, 2023-505764-11-00, 2023
- Acronym
- VISTAS
- Status last verified
- Oct 2026
- First submitted
- 17 Nov 2020
- Last updated
- 19 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No