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
Who is this study for? Patients with Primary Sclerosing Cholangitis
What treatments are being studied? Volixibat
Status: Active_not_recruiting
Location: See all (103) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 12
Healthy Volunteers: f
View:
• 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.
Locations
United States
California
Southern California Research Center
Coronado
Cedars Sinai Medical Center
Los Angeles
University of California, Davis
Sacramento
California Pacific Medical Center Research Institute
San Francisco
Colorado
University of Colorado - Anschutz Medical Campus
Aurora
Washington, D.c.
MedStar Georgetown University Hospital
Washington D.c.
Florida
Florida Research Institute
Lakewood Rch
University of Miami - Schiff Center for Liver Diseases
Miami
Tampa General Hospital
Tampa
Massachusetts
Beth Israel Deaconess Medical Center
Boston
Michigan
Henry Ford Hospital
Detroit
Minnesota
Mayo Clinic
Rochester
Mississippi
Southern Therapy and Advanced Research LLC
Jackson
North Carolina
Duke Health - Duke University Medical Center
Durham
Science 37, Inc (Remote-homebased Telemedicine)
Morrisville
New York
Northwell Health
Manhasset
Columbia University Medical Center - Presbyterian Hospital and Vanderbilt Clinic
New York
New York University Langone Health
New York
Weill Cornell Medical College
New York
University of Rochester Medical Center - Strong Memorial Hospital
Rochester
Ohio
Cleveland Clinic
Cleveland
Pennsylvania
Einstein Healthcare Network - Einstein Medical Center
Philadelphia
University of Pittsburgh Medical Center
Pittsburgh
Tennessee
Galen Medical Group
Hixson
Vanderbilt University Medical Center
Nashville
Texas
The Liver Institute At Methodist Dallas Medical Center
Dallas
University of Texas Southwestern Medical Center
Dallas
Houston Methodist Hospital
Houston
Liver Associates of Texas
Houston
Utah
University of Utah Health Care
Salt Lake City
Virginia
Bon Secours Liver Institution of Hampton Roads Mary Immaculate Hospital Office
Newport News
Richmond Community Hospital LLC
Richmond
Washington
Liver Institute Northwest
Seattle
University of Washington - Harborview Medical Center
Seattle
Other Locations
Argentina
Hospital Italiano de Buenos Aires
Buenos Aires
Glenny Corp S.A.
Ciudad Autónoma Buenos Aires
Hospital El Cruce
San Juan Bautista
Australia
Royal Adelaide Hospital
Adelaide
Northern Health
Epping
Liverpool Hospital
Liverpool
Austin Health
Melbourne
University of the Sunshine Coast
Sippy Downs
Belgium
Cliniques Universitaires Saint-Luc
Brussels
Universitair Ziekenhuis Antwerpen (UZA)
Ghent
University Hospital Ghent
Ghent
Brazil
Hospital das Clinicas da UFMG
Belo Horizonte
Hospital Das Clínicas Da Ufg
Goiânia
Hospital de Clinicas de Porto Alegre
Porto Alegre
Escola Bahiana de Medicina e Saude Publica
Salvador
Hospital do Rim e Hipertensao
São Paulo
Canada
University of Calgary
Calgary
University of Alberta
Edmonton
McMaster University Hospital
Hamilton
London Health Sciences Centre University Hospital
London
McGill University Health Centre - Royal Victoria Hospital
Montreal
The Ottawa Hospital - General Campus
Ottawa
University Health Network - Toronto General Hospital
Toronto
France
Centre Hospitalier Universitaire Grenoble Alpes (CHU Grenoble Alpes)
Grenoble
Centre Hospitalier Universitaire de Lille
Lille
APHP-Hôpital Saint Antoine
Paris
CHU Bordeaux - Haut Lévêque
Pessac
Germany
Charité Universtitätsmedizin - Campus Virchow-Klinikum
Berlin
Universitaetsklinikum Bonn
Bonn
Universitaetsklinikum Erlangen
Erlangen
Hannover Medical School
Hanover
Universitätsklinikum Schleswig-Holstein - Campus Kiel
Kiel
Universitatsklinikum Leipzig
Leipzig
Otto-von-Guericke-Universitat Magdeburg
Magdeburg
University Hospital Tubingen Medical Clinic
Tübingen
St. Josefs-Hospital Wiesbaden
Wiesbaden
Israel
Hillel Yaffe Medical Center
Hadera
Carmel Medical Center
Haifa
Rambam Medical Center
Haifa
Hadassah Medical Center - Ein Karem
Jerusalem
Shaare Zedek Medical Center
Jerusalem
Galilee Medical Center
Nahariya
Rabin Medical Center
Petah Tikva
Sheba Medical Center
Ramat Gan
Tel Aviv Sourasky Medical Center
Tel Aviv
Italy
Azienda Ospedaliero-Universitaria di Bologna - Policlinico S. Orsola-Malpighi
Bologna
ASST Grande Ospedale Metropolitano Niguarda
Milan
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Milan
Fondazione IRCCS San Gerardo dei Tintori
Monza
A.O.U. Federico II
Napoli
Azienda Ospedale Universita Padova
Padua
Netherlands
Amsterdam UMC - VU Medisch Centrum (VUmc)
Amsterdam
Universitair Medisch Centrum Groningen
Groningen
Radboud University Medical Centre (MC)
Nijmegen
Erasmus University Medical Center
Rotterdam
Spain
Hospital Clinic Barcelona
Barcelona
Hospital General Universitario Gregorio Maranon
Madrid
Switzerland
University Hospital Zurich
Zurich
United Kingdom
Royal Victoria Hospital
Belfast
University Hospitals Birmingham NHS Foundation Trust - Wellcome Trust Birmingham Clinical Research Facility
Birmingham
Cambridge University Hospitals NHS Foundation Trust - Addenbrooke's Hospital
Cambridge
Lothian NHS Board
Edinburgh
St James' University Hospital Leeds Teaching Hospitals NHS Trust
Leeds
King's College Hospital NHS Foundation Trust
London
Royal Free Hospital
London
The Newcastle upon Tyne Hospitals NHS Foundation Trust - Royal Victoria Infirmary
Newcastle Upon Tyne
Nottingham University Hospital NHS Trust
Nottingham
John Radcliffe Hospital
Oxford
University Hospital Southampton NHS Foundation Trust
Southampton
Time Frame
Start Date: 2020-12-18
Completion Date: 2027-04
Participants
Target number of participants: 182
Treatments
Experimental: Part 1 Arm 1: Volixibat 20mg
Participants randomized to this arm will receive volixibat 20mg twice daily.
Experimental: Part 1 Arm 2: Volixibat 80mg
Participants randomized to this arm will receive volixibat 80mg twice daily.
Placebo_comparator: Part 1 Arm 3: Placebo
Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
Experimental: Part 2 Arm 1: Volixibat Selected Dose 20mg
Participants randomized to this arm will receive volixibat 20mg twice daily.
Placebo_comparator: Part 2 Arm 2: Placebo
Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
Authors
Vinay Sundaram, Charles Landis, Mordechai Rabinovitz, Michael Porayko, Juan Gallegos-Orozco, Carmen Stanca, Arun Khazanchi, Lisa Forman, Walid Ayoub, Marlyn Mayo, Ira Jacobson, David Bernstein, Cynthia Levy, Kris Kowdley, John Lake, Julius Wilder, Parvez Mantry, Roman Perri, Stuart Gordon, Mitchell Shiffman, Jonathan Huang, Tarek Hassanein, Kidist Yimam, Angela Cheung, Emma Culver, Leona Dold, Janisha Patel, Brian Borg, Alan Bonder, Christopher Bowlus, Gideon Hirschfield
Related Therapeutic Areas
Sponsors
Leads: Mirum Pharmaceuticals, Inc.