RecruitingPhase 2NCT05050136

A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Biliary Cholangitis

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 Biliary Cholangitis


Sponsor

Mirum Pharmaceuticals, Inc.

Enrollment

260 participants

Start Date

Sep 22, 2021

Study Type

INTERVENTIONAL

Conditions

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 Biliary Cholangitis (PBC), and to assess the possible impact on the disease progression of PBC.


Eligibility

Min Age: 18 Years

Inclusion Criteria5

  • Provide signed informed consent at the screening visit as well as comply with all study visits and requirements through the end of the study
  • Male or female, age ≥18 years at the screening visit
  • Confirmed diagnosis of PBC in line with the AASLD guidelines
  • UDCA, anti-pruritic medication and PPAR agonist use will be allowed if meeting additional criteria
  • Qualified pruritus associated with PBC as assessed by Adult ItchRO

Exclusion Criteria6

  • Pruritus associated with an etiology other than PBC
  • Evidence or clinical suspicion of decompensated cirrhosis or a history of decompensation events
  • Current symptomatic cholelithiasis or inflammatory gallbladder disease
  • History of small bowel surgery/resection impacting the terminal ileum that may disrupt the enterohepatic circulation.
  • Evidence, history, or suspicion of other liver diseases; PBC patients with AIH are not excluded
  • History of Liver transplantation

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Interventions

DRUGVolixibat

Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.

DRUGPlacebo

Capsules matched to study drug without the active pharmaceutical ingredient


Locations(130)

Southern California Research Center

Coronado, California, United States

Cedars Sinai Medical Center

Los Angeles, California, United States

University of California Los Angeles

Los Angeles, California, United States

UC Davis

Sacramento, California, United States

University of Colorado

Aurora, Colorado, United States

Yale University Digestive Diseases

New Haven, Connecticut, United States

Covenant Metabolic Specialists

Fort Myers, Florida, United States

UF Hepatology Research at CTRB

Gainesville, Florida, United States

UF Health Gastroenterology- Emerson

Jacksonville, Florida, United States

IHS Health

Kissimmee, Florida, United States

Schiff Center for Liver Diseases

Miami, Florida, United States

Advanced Research Institute, Inc

New Port Richey, Florida, United States

Covenant Research and Clinics, LLC

Sarasota, Florida, United States

Tampa General Hospital

Tampa, Florida, United States

Cleveland Clinic Florida

Weston, Florida, United States

Southeast Clinical Research Center

Dalton, Georgia, United States

Northwestern University

Chicago, Illinois, United States

Loyola University Health System - Loyola Outpatient Center

Maywood, Illinois, United States

University of Iowa Hospitals & Clinics

Iowa City, Iowa, United States

Ochsner Clinic Foundation-Baton Rouge

Baton Rouge, Louisiana, United States

LSU Health Science Center Shreveport

Shreveport, Louisiana, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

Beth Israel Deaconess

Boston, Massachusetts, United States

Mayo Clinic

Rochester, Minnesota, United States

Jackson Liver and GI Specialists

Jackson, Mississippi, United States

CHI Health Clinic (Alegent Creighton Clinic)- Gastroenterology-Bergan

Omaha, Nebraska, United States

Virtua Center for Liver Disease-Camden

Camden, New Jersey, United States

Montefiore Medical Center

The Bronx, New York, United States

Science 37, Inc (Remote-homebased Telemedicine)

Morrisville, North Carolina, United States

Cleveland Clinic - Main Campus

Cleveland, Ohio, United States

Einstein Healthcare Network - Einstein Medical Center

Philadelphia, Pennsylvania, United States

Methodist Le Bonheur Healthcare

Memphis, Tennessee, United States

Galen Medical Group, P.C

Tennessee City, Tennessee, United States

Amel Med LLC

Austin, Texas, United States

The Liver Institute at Methodist Dallas Medical Center

Dallas, Texas, United States

Soma Clinical Trials

Denison, Texas, United States

Baylor College of Medicine

Houston, Texas, United States

Liver Associates of Texas

Houston, Texas, United States

Impact Research

Waco, Texas, United States

University of Utah Health Care

Salt Lake City, Utah, United States

MedStar Health Research Institute

Fairfax, Virginia, United States

Bon Secours Liver Institute of Hampton Roads Mary Immaculate Hospital

Newport News, Virginia, United States

Bon Secours Richmond Community Hospital LLC d/b/a Bon Secours Liver Institute of Richmond

Richmond, Virginia, United States

Liver Institute Northwest

Seattle, Washington, United States

Hospital de Alta Complejidad En Red El Cruce

San Juan Bautista, Buenos Aires, Argentina

Hospital Provincial Del Centenario

Rosario, Santa Fe Province, Argentina

Glenny Corp S.A

CABA, Argentina

Clínica de Nefrología, Urología y Enfermedades Cardiovasculares

Santa Fe, Argentina

Universitair Ziekenhuis Antwerpe UZA

Antwerp, Belgium

Cliniques Universitaires Saint-Luc

Brussels, Belgium

UZ Gent

Ghent, Belgium

Centro de Pesquisa Clínica da Bahiana Saúde

Salvador, Estado de Bahia, Brazil

Hospital Das Clínicas Da Ufg

Goiânia, Goiás, Brazil

Centro de Pesquisas Clínicas do HC/UFMG-EBSERH

Belo Horizonte, Minas Gerais, Brazil

Hospital de Clínicas de Porto Alegre

Porto Alegre, Rio Grande do Sul, Brazil

Hospital Universitário Clementino Fraga Filho - Secretaria de Hepatologia

Rio de Janeiro, Brazil

Instituto de Educação, Pesquisa e Gestão em Saúde

Rio de Janeiro, Brazil

Fundação Oswaldo Ramos

São Paulo, Brazil

Gordon and Leslie Diamond Health Care Centre, Division of Gastroenterology

Vancouver, British Columbia, Canada

(G.I.R.I.) GI Research Institute Foundation

Vancouver, British Columbia, Canada

McMaster University Medical Center

Hamilton, Ontario, Canada

University Hospital, LHSC

London, Ontario, Canada

Toronto General Hospital

Toronto, Ontario, Canada

McGill University Health Centre

Montreal, Quebec, Canada

University of Alberta

Edmonton, Canada

CRCHUM (Centre de Recherche du Centre Hospitalier de l'Université de Montréal)

Montreal, Canada

Mengchao Hepatobiliary Hospital of Fujian Medical University

Fuzhou, Fujian, China

Union Hospital Tongji Medical College Huazhong University of Science and Technology

Wuhan, Hubei, China

The First Hospital of Jilin University

Changchun, Jilin, China

The First Affiliated Hospital, Zhejiang University School of Medicine

Hangzhou, Zhejiang, China

Beijing Youan Hospital, Capital Medical University

Beijing, China

Beijing Friendship Hospital, Capital Medical University

Beijing, China

Sir Run Run Shaw Hospital -Zhejiang University School of Medicine

Hangzhou, China

CHU Grenoble - Hôpital MICHALLON

Grenoble, France

Hopital Claude Huriez-Digestive system disease clinic

Lille, France

CHU de NICE- Hospital L'Archet 2

Nice, France

Hospital Saint Antoine

Paris, France

Nouvel Hospital Civil

Strasbourg, France

Hospital Paul Brousse- APHP

Villejuif, France

Klinikum Chemnitz gGmbH

Chemnitz, Germany

Universitätsklinikum Frankfurt am Main

Frankfurt, Germany

Medizinische Hochschule Hannover (MHH)

Hanover, Germany

Universitätsklinikum Schleswig-Holstein

Kiel, Germany

Universitätsklinikum Leipzig, Klinik und Poliklinik für Onkologie, Gastroenterologie, Hepatologie und Pneumologie

Leipzig, Germany

Otto-von-Guericke-Universität Klinik für Gastroenterologie, Hepatologie und Infektiologie Universitätsklinikum Magdeburg A.ö.R.

Magdeburg, Germany

University Hospital Munster

Münster, Germany

University Hospital Tübingen 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, Liver Institute Kiryat Hadassah POB 12000

Jerusalem, Israel

Galilee Medical Center

Nahariya, Israel

Rabin Medical Center

Petah Tikva, Israel

Sheba Medical Center

Ramat Gan, Israel

The Tel Aviv Sourasky Medical Center

Tel Aviv, Israel

Universita Politecnica delle Marche- Ospedali Riuniti

Ancona, Italy

ASST Papa Giovanni XXIII

Bergamo, Italy

Fondazione Ca Granda

Milan, Italy

ASST Grande Ospedale Metropolitano Niguarda

Milan, Italy

Azienda Ospedaliero Universitaria Di Modena

Modena, Italy

Fondazione IRCCS San Gerardo dei Tintori

Monza, Italy

Azienda Ospedale-Università di Padova

Padova, Italy

Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone

Palermo, Italy

Azienda Ospedaliero Universitaria Pisana (AOUP)

Pisa, Italy

Istituto Clinico Humanitas

Rozzano, Italy

National Hospital Organization Nagasaki Medical Center

Ōmura, Nagasaki, Japan

Ehime University Hospital

Ehime, Japan

Teine Keijinkai Hospital

Hokkaido, Japan

Osaka Rosai Hospital

Osaka, Japan

Toranomon Hospital

Tokyo, Japan

Amstedam University Medical Centers

Amsterdam, Netherlands

Radboudumc

Nijmegen, Netherlands

Erasmus medical center

Rotterdam, Netherlands

Hospital Universitari Vall d' Hebron

Barcelona, Spain

Hospital Clínic de Barcelona

Barcelona, Spain

Hospital Universitari Parc Tauli

Barcelona, Spain

Hospital General Universitario Gregorio Maranon

Madrid, Spain

Hospital Universitario Virgen del Rocio

Seville, Spain

Hospital Universitario Miguel Servet

Zaragoza, Spain

Ente Ospedaliero Cantonale

Lugano, Switzerland

University Hospital Zurich(USZ)

Zurich, Switzerland

University Hospital Southampton NHS Foundation Trust

Southampton, Hampshire, United Kingdom

University Hospitals Birmingham NHS Foundation Trust of Trust Headquarters

Birmingham, West Midlands, United Kingdom

Belfast Health & Social Care Trust (BHSCT) Royal Victoria Hospital

Belfast, United Kingdom

Royal Free Hospital NHS Foundation Trust

London, United Kingdom

King's College Hospital NHS Foundation Trust

London, United Kingdom

Freeman Hospital

Newcastle upon Tyne, United Kingdom

Nottingham University Hospitals NHS Trust

Nottingham, United Kingdom

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