A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC) (VISTAS)

April 15, 2024 updated by: Mirum Pharmaceuticals, Inc.

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

200

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Canada
    • Ontario
      • Ottawa, Ontario, Canada
        • Terminated
        • The Ottawa Hospital - General Campus
      • Toronto, Ontario, Canada, M5G 2C4
        • Recruiting
        • University Health Network - Toronto General Hospital
        • Contact:
        • Principal Investigator:
          • Gideon Hirschfield, MD
  • Germany
      • Berlin, Germany, 13353
        • Recruiting
        • Charité Universtitätsmedizin - Campus Virchow-Klinikum
        • Contact:
        • Principal Investigator:
          • Münevver Demir
      • Erlangen, Germany, 91054
        • Terminated
        • Universitaetsklinikum Erlangen
      • Kiel, Germany, 24105
        • Terminated
        • Universitätsklinikum Schleswig-Holstein - Campus Kiel
      • Leipzig, Germany, 04103
      • Tübingen, Germany, 72076
        • Recruiting
        • University Hospital Tübingen Medical clinic
        • Principal Investigator:
          • Christoph Berg, Prof. Dr. med.
        • Contact:
      • Wiesbaden, Germany, 65189
        • Recruiting
        • St. Josefs-Hospital Wiesbaden
        • Principal Investigator:
          • Christoph Sarrazin, Prof.
        • Contact:
    • Lower Saxony
      • Hannover, Lower Saxony, Germany, 30625
        • Recruiting
        • Hannover Medical School
        • Contact:
        • Principal Investigator:
          • Heiner Wedemeyer, Prof.
    • Nordrhine-Westphalia
      • Bonn, Nordrhine-Westphalia, Germany, 53127
        • Recruiting
        • Universitaetsklinikum Bonn
        • Principal Investigator:
          • Leona Dold, MD
  • Israel
      • Hadera, Israel, 38100
        • Recruiting
        • Hillel Yaffe Medical Center
        • Contact:
        • Principal Investigator:
          • Fadi Abu-Baker
      • Haifa, Israel, 3109601
      • Haifa, Israel, 3434104
        • Recruiting
        • Carmel Medical Center
        • Contact:
        • Principal Investigator:
          • Eli Zuckerman, MD
      • Jerusalem, Israel, 9103102
        • Recruiting
        • Shaare Zedek Medical Center
        • Principal Investigator:
          • Gadi Lalazar, MD
        • Contact:
      • Jerusalem, Israel, 91120
        • Recruiting
        • Hadassah Medical Center - Ein Karem
        • Contact:
        • Principal Investigator:
          • Rifaat Safadi
      • Nahariyya, Israel, 2210001
        • Recruiting
        • Galilee Medical Center
        • Contact:
        • Principal Investigator:
          • Nimer Assy, MD
      • Petach Tikva, Israel, 4941492
        • Recruiting
        • Rabin Medical Center
        • Contact:
        • Principal Investigator:
          • Marius Braun, Dr.
      • Ramat Gan, Israel, 52621
        • Recruiting
        • Sheba Medical Center
        • Contact:
        • Principal Investigator:
          • Ehud Zigmond, Dr.
      • Tel Aviv, Israel, 6423906
        • Terminated
        • Tel Aviv Sourasky Medical Center
  • United Kingdom
      • Belfast, United Kingdom, BT12 6BA
      • Birmingham, United Kingdom, B15 2TH
        • Recruiting
        • University Hospitals Birmingham NHS Foundation Trust - Wellcome Trust Birmingham Clinical Research Facility
        • Contact:
        • Principal Investigator:
          • Palak Trivedi
      • Cambridge, United Kingdom, CB2 0QQ
        • Recruiting
        • Cambridge University Hospitals NHS Foundation Trust - Addenbrooke's Hospital
        • Contact:
        • Principal Investigator:
          • George Mells
      • Edinburgh, United Kingdom, EH164SA
      • Leeds, United Kingdom, LS9 7TF
        • Terminated
        • St James' University Hospital Leeds Teaching Hospitals NHS Trust
      • London, United Kingdom, SE5 9RS
        • Recruiting
        • King's College Hospital NHS Foundation Trust
        • Principal Investigator:
          • Deepask Joshi, MD
        • Contact:
      • Nottingham, United Kingdom, NG7 2FT
        • Recruiting
        • Nottingham University Hospital NHS Trust
        • Contact:
        • Principal Investigator:
          • Suresh V Venkatachalapathy
      • Southampton, United Kingdom
        • Recruiting
        • University Hospital Southampton NHS Foundation Trust
        • Principal Investigator:
          • Janisha Patel, MD
        • Contact:
    • Hampstead
      • London, Hampstead, United Kingdom, NW3 2QG
    • Headington
      • Oxford, Headington, United Kingdom, OX3 9DU
        • Recruiting
        • John Radcliffe Hospital
        • Principal Investigator:
          • Emma Culver, MD
        • Contact:
  • United States
    • California
      • Coronado, California, United States, 92118
        • Recruiting
        • Southern California Research Center
        • Contact:
        • Principal Investigator:
          • Tarek Hassanein, MD
      • Culver City, California, United States, 90230
        • Recruiting
        • Science 37, Inc (Remote-homebased Telemedicine)
        • Contact:
        • Principal Investigator:
          • Debra L. Weinstein, MD, CPI
      • Los Angeles, California, United States, 90048
        • Recruiting
        • Cedars Sinai Medical Center
        • Principal Investigator:
          • Walid Ayoub, MD
        • Contact:
      • Sacramento, California, United States, 95817
        • Recruiting
        • University of California, Davis
        • Principal Investigator:
          • Christopher Bowlus, MD
        • Contact:
      • San Francisco, California, United States, 94109
        • Recruiting
        • California Pacific Medical Center Research Institute
        • Principal Investigator:
          • Kidist Yimam, MD
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Recruiting
        • University of Colorado - Anschutz Medical Campus
        • Principal Investigator:
          • Lisa Forman, MD
        • Contact:
    • District of Columbia
      • Washington, District of Columbia, United States, 20007
        • Recruiting
        • MedStar Georgetown University Hospital
        • Principal Investigator:
          • Coleman Smith
        • Contact:
    • Florida
      • Lakewood Ranch, Florida, United States, 34211
        • Terminated
        • Florida Research Institute
      • Miami, Florida, United States, 33136
        • Recruiting
        • University of Miami - Schiff Center for Liver Diseases
        • Contact:
        • Principal Investigator:
          • Cynthia Levy, MD
      • Tampa, Florida, United States, 33606
        • Recruiting
        • Tampa General Hospital
        • Principal Investigator:
          • Nyingi Kemmer
        • Contact:
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
        • Recruiting
        • Beth Israel Deaconess Medical Center
        • Principal Investigator:
          • Alan Bonder, MD
        • Contact:
    • Michigan
      • Detroit, Michigan, United States, 48202
        • Recruiting
        • Henry Ford Hospital
        • Principal Investigator:
          • Stuart Gordon, MD
        • Contact:
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Recruiting
        • Mayo Clinic
        • Contact:
        • Principal Investigator:
          • John Eaton, MD
    • Mississippi
      • Jackson, Mississippi, United States, 39216
        • Terminated
        • Southern Therapy and Advanced Research LLC
    • New York
      • Manhasset, New York, United States, 11030
        • Terminated
        • Northwell Health
      • New York, New York, United States, 10021
        • Recruiting
        • Weill Cornell Medical College
        • Principal Investigator:
          • Emily Schonfeld, MD
        • Contact:
      • New York, New York, United States, 10016
      • New York, New York, United States, 10032
        • Recruiting
        • Columbia University Irving Medical Center - Research Pharmacy
        • Contact:
        • Principal Investigator:
          • Mercedes Martinez
      • Rochester, New York, United States, 14642
        • Terminated
        • University of Rochester Medical Center - Strong Memorial Hospital
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Recruiting
        • Duke Health - Duke University Medical Center
        • Principal Investigator:
          • Julius Wilder, MD
        • Contact:
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Recruiting
        • Cleveland Clinic
        • Contact:
        • Principal Investigator:
          • Olumuyiwa Awoniyi, MD
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19141
        • Recruiting
        • Einstein Healthcare Network - Einstein Medical Center
        • Principal Investigator:
          • Manish Thapar, MD
        • Contact:
      • Pittsburgh, Pennsylvania, United States, 15213
        • Terminated
        • University of Pittsburgh Medical Center
    • Tennessee
      • Hixson, Tennessee, United States, 37343
        • Recruiting
        • Galen Medical Group
        • Contact:
        • Principal Investigator:
          • Chirag Patel
      • Nashville, Tennessee, United States, 37232
        • Recruiting
        • Vanderbilt University Medical Center
        • Principal Investigator:
          • Roman Perri, MD
        • Contact:
    • Texas
      • Dallas, Texas, United States, 75390
      • Dallas, Texas, United States, 75203
        • Recruiting
        • The Liver Institute At Methodist Dallas Medical Center
        • Contact:
        • Principal Investigator:
          • Parvez Mantry, MD
      • Houston, Texas, United States, 77030
        • Recruiting
        • Liver Associates of Texas
        • Principal Investigator:
          • Victor Ankoma-Sey
        • Contact:
      • Houston, Texas, United States, 77030
        • Terminated
        • Houston Methodist Hospital
    • Utah
      • Salt Lake City, Utah, United States, 84132
        • Recruiting
        • University of Utah Health Care
        • Principal Investigator:
          • Juan Gallegos-Orozco, MD
        • Contact:
    • Virginia
      • Newport News, Virginia, United States, 23603
        • Recruiting
        • Bon Secours Liver Institution of Hampton Roads Mary Immaculate Hospital Office
        • Contact:
        • Principal Investigator:
          • Mitchell Shiffman, MD
      • Richmond, Virginia, United States, 23226
        • Recruiting
        • Richmond Community Hospital LLC
        • Contact:
        • Principal Investigator:
          • Mitchell Shiffman, MD
    • Washington
      • Seattle, Washington, United States, 98105
      • Seattle, Washington, United States, 98104
        • Terminated
        • University of Washington - Harborview Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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 diseases
  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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Volixibat 20mg
Participants randomized to this arm will receive volixibat 20mg twice daily.
Drug: Volixibat
Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.
Other Names:
  • SHP626
Experimental: Volixibat 80mg
Participants randomized to this arm will receive volixibat 80mg twice daily.
Drug: Volixibat
Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.
Other Names:
  • SHP626
Placebo Comparator: Placebo
Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
Drug: Placebo
Capsules matched to study drug minus active substance

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire
Time Frame: Baseline through to Week 28
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
Proportion of participants with itch response using the Adult ItchRO
Time Frame: Baseline through to Week 28
Baseline through to Week 28
The incidence of adverse events
Time Frame: Baseline through to Week 28
Baseline through to Week 28
Changes in alkaline phosphatase
Time Frame: Baseline through to Week 28
Baseline through to Week 28
Change in Primary Sclerosing Cholangitis-Specific Patient-Reported Outcome
Time Frame: Baseline through to Week 28
Baseline through to Week 28
Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) fatigue questionnaire
Time Frame: Baseline through to Week 28
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.
Baseline through to Week 28
Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) sleep disturbance questionnaire
Time Frame: Baseline through to Week 28
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.
Baseline through to Week 28
Changes in serum bile acid levels
Time Frame: Baseline through to Week 28
Baseline through to Week 28
Changes in total bilirubin levels
Time Frame: Baseline through to Week 28
Baseline through to Week 28

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Mirum Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 18, 2020

Primary Completion (Estimated)

September 1, 2025

Study Completion (Estimated)

December 1, 2025

Study Registration Dates

First Submitted

November 17, 2020

First Submitted That Met QC Criteria

December 9, 2020

First Posted (Actual)

December 11, 2020

Study Record Updates

Last Update Posted (Actual)

April 17, 2024

Last Update Submitted That Met QC Criteria

April 15, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VLX-301
  • 2020-003027-41 (EudraCT Number)
  • 2023-505764-11-00 (Other Identifier: EUCT number)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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