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

NCT ID: NCT05050136

Last Updated: 2026-01-20

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

260 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-09-22

Study Completion Date

2027-07-31

Brief Summary

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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.

Detailed Description

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Conditions

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Primary Biliary Cholangitis PBC

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Part 1 Arm 1: Volixibat 20mg

Participants randomized to this arm will receive volixibat 20mg twice daily.

Group Type EXPERIMENTAL

Volixibat

Intervention Type DRUG

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

Part 1 Arm 2: Volixibat 80mg

Participants randomized to this arm will receive volixibat 80mg twice daily.

Group Type EXPERIMENTAL

Volixibat

Intervention Type DRUG

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

Part 1 Arm 3: Placebo

Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Capsules matched to study drug without the active pharmaceutical ingredient

Part 2 Arm 1: Volixibat Selected Dose 20mg

Participants randomized to this arm will receive volixibat 20mg twice daily.

Group Type EXPERIMENTAL

Volixibat

Intervention Type DRUG

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

Part 2 Arm 2: Placebo

Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Capsules matched to study drug without the active pharmaceutical ingredient

Interventions

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Placebo

Capsules matched to study drug without the active pharmaceutical ingredient

Intervention Type DRUG

Volixibat

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

Intervention Type DRUG

Other Intervention Names

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SHP626

Eligibility Criteria

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Inclusion Criteria

* 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 Criteria

* 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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Mirum Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Southern California Research Center

Coronado, California, United States

Site Status TERMINATED

Cedars Sinai Medical Center

Los Angeles, California, United States

Site Status RECRUITING

UC Davis

Sacramento, California, United States

Site Status RECRUITING

University of Colorado

Aurora, Colorado, United States

Site Status RECRUITING

Yale University Digestive Diseases

New Haven, Connecticut, United States

Site Status RECRUITING

Covenant Metabolic Specialists

Fort Myers, Florida, United States

Site Status RECRUITING

UF Hepatology Research at CTRB

Gainesville, Florida, United States

Site Status RECRUITING

UF Health Gastroenterology- Emerson

Jacksonville, Florida, United States

Site Status RECRUITING

IHS Health

Kissimmee, Florida, United States

Site Status TERMINATED

Schiff Center for Liver Diseases

Miami, Florida, United States

Site Status RECRUITING

Advanced Research Institute, Inc

New Port Richey, Florida, United States

Site Status RECRUITING

Covenant Research and Clinics, LLC

Sarasota, Florida, United States

Site Status RECRUITING

Tampa General Hospital

Tampa, Florida, United States

Site Status RECRUITING

Southeast Clinical Research Center

Dalton, Georgia, United States

Site Status RECRUITING

Northwestern University

Chicago, Illinois, United States

Site Status RECRUITING

Loyola University Health System - Loyola Outpatient Center

Maywood, Illinois, United States

Site Status RECRUITING

University of Iowa Hospitals & Clinics

Iowa City, Iowa, United States

Site Status RECRUITING

Ochsner Clinic Foundation-Baton Rouge

Baton Rouge, Louisiana, United States

Site Status RECRUITING

LSU Health Science Center Shreveport

Shreveport, Louisiana, United States

Site Status RECRUITING

Beth Israel Deaconess

Boston, Massachusetts, United States

Site Status RECRUITING

Mayo Clinic

Rochester, Minnesota, United States

Site Status RECRUITING

Jackson Liver and GI Specialists

Jackson, Mississippi, United States

Site Status TERMINATED

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

Omaha, Nebraska, United States

Site Status RECRUITING

Science 37, Inc (Remote-homebased Telemedicine)

Morrisville, North Carolina, United States

Site Status RECRUITING

Cleveland Clinic - Main Campus

Cleveland, Ohio, United States

Site Status RECRUITING

Einstein Healthcare Network - Einstein Medical Center

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Methodist Le Bonheur Healthcare

Memphis, Tennessee, United States

Site Status TERMINATED

Galen Medical Group, P.C

Tennessee City, Tennessee, United States

Site Status RECRUITING

Amel Med LLC

Austin, Texas, United States

Site Status RECRUITING

Soma Clinical Trials

Denison, Texas, United States

Site Status RECRUITING

Baylor College of Medicine

Houston, Texas, United States

Site Status RECRUITING

Liver Associates of Texas

Houston, Texas, United States

Site Status RECRUITING

Velocity Clinical Research

Waco, Texas, United States

Site Status RECRUITING

University of Utah Health Care

Salt Lake City, Utah, United States

Site Status RECRUITING

MedStar Health Research Institute

Fairfax, Virginia, United States

Site Status RECRUITING

Bon Secours Liver Institute of Hampton Roads Mary Immaculate Hospital

Newport News, Virginia, United States

Site Status RECRUITING

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

Richmond, Virginia, United States

Site Status RECRUITING

Liver Institute Northwest

Seattle, Washington, United States

Site Status RECRUITING

Hospital de Alta Complejidad En Red El Cruce

San Juan Bautista, Buenos Aires, Argentina

Site Status RECRUITING

Hospital Provincial Del Centenario

Rosario, Santa Fe Province, Argentina

Site Status RECRUITING

Glenny Corp S.A

CABA, , Argentina

Site Status RECRUITING

Universitair Ziekenhuis Antwerpe UZA

Antwerp, , Belgium

Site Status RECRUITING

Cliniques Universitaires Saint-Luc

Brussels, , Belgium

Site Status RECRUITING

UZ Gent

Ghent, , Belgium

Site Status RECRUITING

Centro de Pesquisa Clínica da Bahiana Saúde

Salvador, Estado de Bahia, Brazil

Site Status RECRUITING

Hospital Das Clínicas Da Ufg

Goiânia, Goiás, Brazil

Site Status RECRUITING

Centro de Pesquisas Clínicas do HC/UFMG-EBSERH

Belo Horizonte, Minas Gerais, Brazil

Site Status RECRUITING

Hospital de Clínicas de Porto Alegre

Porto Alegre, Rio Grande do Sul, Brazil

Site Status RECRUITING

Fundação Oswaldo Ramos

São Paulo, , Brazil

Site Status RECRUITING

Gordon and Leslie Diamond Health Care Centre, Division of Gastroenterology

Vancouver, British Columbia, Canada

Site Status RECRUITING

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

Vancouver, British Columbia, Canada

Site Status RECRUITING

McMaster University Medical Center

Hamilton, Ontario, Canada

Site Status RECRUITING

University Hospital, LHSC

London, Ontario, Canada

Site Status RECRUITING

McGill University Health Centre

Montreal, Quebec, Canada

Site Status RECRUITING

University of Alberta

Edmonton, , Canada

Site Status RECRUITING

Mengchao Hepatobiliary Hospital of Fujian Medical University

Fuzhou, Fujian, China

Site Status RECRUITING

Union Hospital Tongji Medical College Huazhong University of Science and Technology

Wuhan, Hubei, China

Site Status RECRUITING

The First Hospital of Jilin University

Changchun, Jilin, China

Site Status RECRUITING

The First Affiliated Hospital, Zhejiang University School of Medicine

Hangzhou, Zhejiang, China

Site Status RECRUITING

Beijing Youan Hospital, Capital Medical University

Beijing, , China

Site Status RECRUITING

Beijing Friendship Hospital, Capital Medical University

Beijing, , China

Site Status RECRUITING

Sir Run Run Shaw Hospital -Zhejiang University School of Medicine

Hangzhou, , China

Site Status RECRUITING

CHU Grenoble - Hôpital MICHALLON

Grenoble, , France

Site Status RECRUITING

Hopital Claude Huriez-Digestive system disease clinic

Lille, , France

Site Status RECRUITING

CHU de NICE- Hospital L'Archet 2

Nice, , France

Site Status RECRUITING

Hospital Saint Antoine

Paris, , France

Site Status RECRUITING

Nouvel Hospital Civil

Strasbourg, , France

Site Status RECRUITING

Hospital Paul Brousse- APHP

Villejuif, , France

Site Status RECRUITING

Klinikum Chemnitz gGmbH

Chemnitz, , Germany

Site Status RECRUITING

Universitätsklinikum Frankfurt am Main

Frankfurt, , Germany

Site Status RECRUITING

Medizinische Hochschule Hannover (MHH)

Hanover, , Germany

Site Status RECRUITING

Universitätsklinikum Schleswig-Holstein

Kiel, , Germany

Site Status RECRUITING

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

Leipzig, , Germany

Site Status RECRUITING

University Hospital Munster

Münster, , Germany

Site Status RECRUITING

University Hospital Tübingen Medical clinic

Tübingen, , Germany

Site Status RECRUITING

St. Josefs-Hospital Wiesbaden

Wiesbaden, , Germany

Site Status RECRUITING

Hillel Yaffe Medical Center

Hadera, , Israel

Site Status RECRUITING

Rambam Medical Center

Haifa, , Israel

Site Status RECRUITING

Carmel Medical Center

Haifa, , Israel

Site Status RECRUITING

Shaare Zedek Medical Center

Jerusalem, , Israel

Site Status TERMINATED

Hadassah Medical Center - Ein Karem, Liver Institute Kiryat Hadassah POB 12000

Jerusalem, , Israel

Site Status RECRUITING

Galilee Medical Center

Nahariya, , Israel

Site Status RECRUITING

Rabin Medical Center

Petah Tikva, , Israel

Site Status RECRUITING

Sheba Medical Center

Ramat Gan, , Israel

Site Status RECRUITING

The Tel Aviv Sourasky Medical Center

Tel Aviv, , Israel

Site Status TERMINATED

Universita Politecnica delle Marche- Ospedali Riuniti

Ancona, , Italy

Site Status RECRUITING

ASST Papa Giovanni XXIII

Bergamo, , Italy

Site Status RECRUITING

Fondazione Ca Granda

Milan, , Italy

Site Status RECRUITING

ASST Grande Ospedale Metropolitano Niguarda

Milan, , Italy

Site Status RECRUITING

Fondazione IRCCS San Gerardo dei Tintori

Monza, , Italy

Site Status RECRUITING

Azienda Ospedale-Università di Padova

Padua, , Italy

Site Status RECRUITING

Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone

Palermo, , Italy

Site Status RECRUITING

Azienda Ospedaliero Universitaria Pisana (AOUP)

Pisa, , Italy

Site Status RECRUITING

Istituto Clinico Humanitas

Rozzano, , Italy

Site Status RECRUITING

National Hospital Organization Nagasaki Medical Center

Ōmura, Nagasaki, Japan

Site Status RECRUITING

Ehime University Hospital

Ehime, , Japan

Site Status RECRUITING

Teine Keijinkai Hospital

Hokkaido, , Japan

Site Status RECRUITING

Osaka Rosai Hospital

Osaka, , Japan

Site Status RECRUITING

Toranomon Hospital

Tokyo, , Japan

Site Status RECRUITING

Amstedam University Medical Centers

Amsterdam, , Netherlands

Site Status RECRUITING

Radboudumc

Nijmegen, , Netherlands

Site Status RECRUITING

Erasmus medical center

Rotterdam, , Netherlands

Site Status RECRUITING

Hospital Universitari Vall d' Hebron

Barcelona, , Spain

Site Status RECRUITING

Hospital Clínic de Barcelona

Barcelona, , Spain

Site Status RECRUITING

Hospital Universitari Parc Tauli

Barcelona, , Spain

Site Status RECRUITING

Hospital General Universitario Gregorio Maranon

Madrid, , Spain

Site Status RECRUITING

Hospital Universitario Virgen del Rocio

Seville, , Spain

Site Status RECRUITING

Hospital Universitario Miguel Servet

Zaragoza, , Spain

Site Status RECRUITING

Ente Ospedaliero Cantonale

Lugano, , Switzerland

Site Status RECRUITING

University Hospital Zurich(USZ)

Zurich, , Switzerland

Site Status RECRUITING

University Hospital Southampton NHS Foundation Trust

Southampton, Hampshire, United Kingdom

Site Status RECRUITING

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

Belfast, , United Kingdom

Site Status RECRUITING

Royal Free Hospital NHS Foundation Trust

London, , United Kingdom

Site Status RECRUITING

King's College Hospital NHS Foundation Trust

London, , United Kingdom

Site Status RECRUITING

Nottingham University Hospitals NHS Trust

Nottingham, , United Kingdom

Site Status RECRUITING

Countries

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United States Argentina Belgium Brazil Canada China France Germany Israel Italy Japan Netherlands Spain Switzerland United Kingdom

Facility Contacts

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Nirosha Tilakaratna

Role: primary

310-423-1269

Kevin Yashar

Role: backup

310-423-2743

Sandeep Dhaliwal

Role: primary

916-734-8696

Charles Stasa

Role: primary

303-724-6875

Suzie Christopher

Role: primary

203-785-4070

Guy Neff, MD

Role: primary

941-500-3200

Julia Surow

Role: backup

941-500-3200

Lasheaka McClellan

Role: primary

352-273-7285

Maged Ghali, MD

Role: primary

904-633-0070

Kelly Jackman

Role: backup

904-633-0070

Edisson Jesus Aguilera

Role: primary

305-243-4648

Christina Tyson

Role: primary

727-835-3261

Veronica Valle

Role: primary

941-500-3200

Jennifer Schmidt, RN, BSN

Role: primary

813-347-2878

Alma Carmona

Role: primary

706-913-1779

Jeanne Gottstein

Role: primary

312-694-0264

Vira Boichuk

Role: primary

708-216-3268

Michelle Kudulis

Role: backup

708-216-0291

Jena Valestin, RN, BSN

Role: primary

319-353-4574

Magen Parker

Role: primary

225-791-5278

Robyne Champagne

Role: primary

318-675-5803

Julie Shea

Role: primary

617-632-1125

Mitchell Clayton

Role: primary

507-284-2698

Madeline Esch

Role: primary

402-343-8511

Clinical Research Coordinator

Role: primary

888-462-7559

Kevin Kruszewski

Role: primary

216-636-5555

Erin McMullen

Role: backup

216-636-5555

Laura Clark

Role: primary

215-456-8721

Kim Norwood

Role: primary

423-954-9015

Sydney Boles

Role: backup

423-954-9015

Meghan Smith, MD

Role: primary

512-718-7415

Mohammed Hassan

Role: primary

469-819-0228

Youseph Karouni

Role: primary

713-798-3706

Quynh-An Phan

Role: primary

713-799-2667

Nancy Canada Canada

Role: primary

254-294-4780

Amy Holman

Role: primary

801-538-0689

Grace Jin

Role: primary

202-877-6501

Naketa Bartlett

Role: primary

757-947-3197

Laura Joines

Role: primary

804-977-8921

Theresa Dorrian

Role: primary

206-536-3030

Mariana Ambrosis

Role: primary

221- 616-5547

Gabriela Juaristi

Role: primary

+54 9 3416 28-5257

Maria Belen Umina

Role: primary

54 911 3086-4247

Sven M Francque

Role: primary

+32-03-821 3323

Umuhire Fabiola

Role: primary

3227641445

Lien Mahy

Role: primary

+32 9 332 94 59

Paulo Bittencourt

Role: primary

55 71 3276-8232

Rodrigo Sebba Aires

Role: primary

55 62 98403-4012

Claudia Couto

Role: primary

(31) 3307-9255

Mário Reis Álvares-da-Silva

Role: primary

51 3359 8749

Maria Lucia Ferraz

Role: primary

(11) 5087-8113

Rosetti Lin

Role: primary

604-875-4111 ext. 61271

Araceli Wilkinson

Role: primary

604-688-6332 ext. 293

Gail Cappelli

Role: primary

905- 570-2100 ext. 76986

Rokhsana Mortuza

Role: primary

519-685-8500 ext. 36893

Tianyan Chen

Role: primary

514-934-1934

Aldo Montano-Loza

Role: primary

780-492-8602

Zu-Xiong Huang

Role: primary

13599395269

Ling Yang

Role: primary

13971178791

Qinglong Jin

Role: primary

0431 88783330

Jifang Sheng

Role: primary

13600519200

Lishan Zhang

Role: primary

15175833690

Lintong Xu

Role: primary

13261887129

Siyi Ying

Role: primary

18258586785

Emma Vige

Role: primary

33 4 76 76 67 45

Chadly Charron

Role: primary

03.20.44.57.42

Katia De Cuyper

Role: primary

33 4 92 03 66 31

Farid Gaouar

Role: primary

33-1-71-97-01-17

Pauline Simo-Noumbissie

Role: primary

33-3 69 55 16 80

Roua Ben Hassine

Role: primary

33 1 45 59 64 23

Jacqueline Uhle

Role: primary

+49 (0)371-333 35631

Role: backup

+49 (0)371-333 33236

Laura Gesell

Role: primary

069-6301-87769

Julia Schneider

Role: primary

+49 511 532 3779

Sonja Rohweder

Role: primary

+4943150022406

Vanessa Maerz

Role: backup

+4943150022406

Thomas Berg

Role: primary

0049 341-97 12330

Christel Langenstroer

Role: primary

0251 8356226

Petra Janke-Maier

Role: primary

+ 49 7071 29-82130

Johanna Bedford

Role: primary

+49 611 177 4271

Elisabeth Mahajna

Role: primary

972-4-7744695

Rimma Kramsky

Role: primary

+972-50-2063188

Vered Ben-Hakoon

Role: backup

+972-50-2061650

Hen Biton

Role: primary

+972-4-8250052

Orly Azulay

Role: backup

+972-4-8250685

Rana Marzeeq

Role: primary

972-52-5128438

Reem Kaloti Dakkak

Role: backup

972-54-3059284

Nelly Bar Guy

Role: primary

+972-527476202

Daniel Ofri

Role: primary

+972-3-652-5104

Hadar Jenin

Role: primary

+972-35307179

Marco Marzioni

Role: primary

+39 34933146731

Pietro Giani

Role: primary

+39 0352673448

Roberta Soffredini

Role: primary

0250320378

Federico D'Amico

Role: primary

+390264448442

Gabriella Marsoni

Role: primary

+390392333962

Emanuela Bonaiuto

Role: primary

+39-3385935024

Vincenza Calvaruso

Role: primary

+39-091-655-1111

Cristina Dettori

Role: primary

050996857

Ilaria Sogno

Role: primary

00390282247319

Atsumasa Komori

Role: primary

81-957-52-3121

Kaori Ikeda

Role: primary

81-80-8049-9030

Kunihiko Tsuji

Role: primary

81-11-681-8111

Emiko Tanaka

Role: primary

81-72-252-3561

Yoshiko Okada

Role: primary

81-3-3588-1111 ext. 3410

Martine Bakker-Peters

Role: primary

020-44 42248

Eva Smit

Role: primary

0031 24 3619190

Milko Van Langen

Role: primary

+31 6 11 03 74 28

Maria del Mar Riveiro Barciela

Role: primary

932746000 ext. 6037

Cristina Ribera

Role: primary

0034 693238732

Iris Lioba Matilla Vaz

Role: primary

93.723.10.10 ext. 80816

Paloma Fernández Cáceres

Role: primary

0034 917996062

Javier Ampuero Herrojo

Role: primary

34-95-5012065

Vanesa Bernal Monterde

Role: primary

34-976-76-55-00

Benedetta Terziroli

Role: primary

41918117635

Andreas Kremer

Role: primary

41-44-255 11 11

Sameerah Battad

Role: primary

023 8120 4149

Heather Lawther

Role: primary

02896150497

Douglas Thorburn

Role: primary

02077940500 ext. 36939

Andrew Ayers

Role: primary

020322997148

Elizabeth Williamson

Role: primary

+44 1159249924 ext. 83711

References

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Klatzkow H, Bhavsar-Burke I, Pearson M, Wentworth BJ. Primary Biliary Cholangitis in 2025: A New Frontier. Am J Gastroenterol. 2025 Dec 1;120(12):2746-2749. doi: 10.14309/ajg.0000000000003559. Epub 2025 May 29. No abstract available.

Reference Type DERIVED
PMID: 40439725 (View on PubMed)

Other Identifiers

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VLX-601

Identifier Type: -

Identifier Source: org_study_id

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