Clinical trial NCT05050136: A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Biliary Cholangitis
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 Biliary Cholangitis
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Primary Biliary Cholangitis
PBC
Keywords: Pruritus
PBC
Itching
Cholangitis
Primary Biliary Cholangitis
Hepatobiliary disorders
Lead sponsor: Mirum Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2021-09-20
Enrollment: 180
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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 criteria: 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 and anti-pruritic medication 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

Primary outcomes:
measure: Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire
timeFrame: Baseline to week 28
Secondary outcomes:
measure: Proportion of participants with itch response using the Adult ItchRO
timeFrame: Baseline to week 28
measure: Incidence of adverse events
timeFrame: Baseline to week 28
measure: Changes in alkaline phosphatase
timeFrame: Baseline to week 28
measure: Changes in total bilirubin
timeFrame: Baseline to week 28
measure: Changes in serum bile acid levels
timeFrame: Baseline to week 28
measure: Change in Primary Biliary Cholangitis Quality of Life Measure (PBC-40)
timeFrame: Baseline to week 28
measure: Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) fatigue questionnaire
timeFrame: Baseline to week 28
measure: Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) sleep disturbance questionnaire
timeFrame: Baseline to week 28
Other outcomes:
Locations:
city: Coronado
country: United States
facility: Southern California Research Center
state: California
city: Los Angeles
country: United States
facility: Cedars Sinai Medical Center
state: California
city: Aurora
country: United States
facility: University of Colorado
state: Colorado
city: Fort Myers
country: United States
facility: Covenant Metabolic Specialists
state: Florida
city: Gainesville
country: United States
facility: UF Hepatology Research at CTRB
state: Florida
city: Jacksonville
country: United States
facility: UF Health Gastroenterology- Emerson
state: Florida
city: Kissimmee
country: United States
facility: IHS Health
state: Florida
city: Miami
country: United States
facility: Schiff Center for Liver Diseases
state: Florida
city: New Port Richey
country: United States
facility: Advanced Research Institute, Inc
state: Florida
city: Sarasota
country: United States
facility: Covenant Research and Clinics, LLC
state: Florida
city: Tampa
country: United States
facility: Tampa General Hospital
state: Florida
city: Dalton
country: United States
facility: Southeast Clinical Research Center
state: Georgia
city: Chicago
country: United States
facility: Northwestern University
state: Illinois
city: Maywood
country: United States
facility: Loyola University Health System - Loyola Outpatient Center
state: Illinois
city: Iowa City
country: United States
facility: University of Iowa Hospitals & Clinics
state: Iowa
city: Baton Rouge
country: United States
facility: Ochsner Clinic Foundation-Baton Rouge
state: Louisiana
city: Shreveport
country: United States
facility: LSU Health Science Center Shreveport
state: Louisiana
city: Boston
country: United States
facility: Beth Israel Deaconess
state: Massachusetts
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Jackson
country: United States
facility: Jackson Liver and GI Specialists
state: Mississippi
city: Omaha
country: United States
facility: CHI Health Clinic (Alegent Creighton Clinic)- Gastroenterology-Bergan
state: Nebraska
city: Morrisville
country: United States
facility: Science 37, Inc (Remote-homebased Telemedicine)
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic - Main Campus
state: Ohio
city: Philadelphia
country: United States
facility: Einstein Healthcare Network - Einstein Medical Center
state: Pennsylvania
city: Memphis
country: United States
facility: Methodist Le Bonheur Healthcare
state: Tennessee
city: Tennessee
country: United States
facility: Galen Medical Group, P.C
state: Tennessee
city: Austin
country: United States
facility: Amel Med LLC
state: Texas
city: Denison
country: United States
facility: Soma Clinical Trials
state: Texas
city: Houston
country: United States
facility: Liver Associates of Texas
state: Texas
city: Waco
country: United States
facility: Velocity Clinical Research
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah Health Care
state: Utah
city: Fairfax
country: United States
facility: MedStar Health Research Institute
state: Virginia
city: Newport News
country: United States
facility: Bon Secours Liver Institute of Hampton Roads Mary Immaculate Hospital
state: Virginia
city: Richmond
country: United States
facility: Bon Secours Richmond Community Hospital LLC d/b/a Bon Secours Liver Institute of Richmond
state: Virginia
city: Seattle
country: United States
facility: Liver Institute Northwest
state: Washington
city: Florencio Varela
country: Argentina
facility: Hospital de Alta Complejidad En Red El Cruce
state: Buenos Aires
city: Caba
country: Argentina
facility: Glenny Corp S.A
city: Antwerp
country: Belgium
facility: Universitair Ziekenhuis Antwerpe UZA
city: Brussel
country: Belgium
facility: Cliniques Universitaires Saint-Luc
city: Gent
country: Belgium
facility: UZ Gent
city: Salvador
country: Brazil
facility: Centro de Pesquisa Clínica da Bahiana Saúde
state: Bahia
city: Goiânia
country: Brazil
facility: Hospital Das Clínicas Da Ufg
state: Goiás
city: Belo Horizonte
country: Brazil
facility: Centro de Pesquisas Clínicas do HC/UFMG-EBSERH
state: Minas Gerais
city: Porto Alegre
country: Brazil
facility: Hospital de Clínicas de Porto Alegre
state: RS
city: São Paulo
country: Brazil
facility: Fundação Oswaldo Ramos
city: Vancouver
country: Canada
facility: Gordon and Leslie Diamond Health Care Centre, Division of Gastroenterology
state: British Columbia
city: London
country: Canada
facility: University Hospital, LHSC
state: Ontario
city: Edmonton
country: Canada
facility: University of Alberta
city: Grenoble
country: France
facility: CHU Grenoble - Hôpital MICHALLON
city: Lille
country: France
facility: Hopital Claude Huriez-Digestive system disease clinic
city: Nice
country: France
facility: CHU de NICE- Hospital L'Archet 2
city: Paris
country: France
facility: Hospital Saint Antoine
city: Strasbourg
country: France
facility: Nouvel Hospital Civil
city: Villejuif
country: France
facility: Hospital Paul Brousse- APHP
city: Chemnitz
country: Germany
facility: Klinikum Chemnitz gGmbH
city: Frankfurt
country: Germany
facility: Universitätsklinikum Frankfurt am Main
city: Hanover
country: Germany
facility: Medizinische Hochschule Hannover (MHH)
city: Kiel
country: Germany
facility: Universitätsklinikum Schleswig-Holstein
city: Münster
country: Germany
facility: University Hospital Munster
city: Tubingen
country: Germany
facility: University Hospital Tübingen Medical clinic
city: Wiesbaden
country: Germany
facility: St. Josefs-Hospital Wiesbaden
city: Hadera
country: Israel
facility: Hillel Yaffe Medical Center
city: Haifa
country: Israel
facility: Rambam Medical Center
city: Haifa
country: Israel
facility: Carmel Medical Center
city: Jerusalem
country: Israel
facility: Shaare Zedek Medical Center
city: Jerusalem
country: Israel
facility: Hadassah Medical Center - Ein Karem, Liver Institute Kiryat Hadassah POB 12000
city: Nahariyya
country: Israel
facility: Galilee Medical Center
city: Petach Tikva
country: Israel
facility: Rabin Medical Center
city: Ramat Gan
country: Israel
facility: Sheba Medical Center
city: Tel Aviv
country: Israel
facility: The Tel Aviv Sourasky Medical Center
city: Ancona
country: Italy
facility: Universita Politecnica delle Marche- Ospedali Riuniti
city: Milano
country: Italy
facility: Fondazione Ca Granda
city: Milano
country: Italy
facility: ASST Grande Ospedale Metropolitano Niguarda
city: Monza
country: Italy
facility: Fondazione IRCCS San Gerardo dei Tintori
city: Padova
country: Italy
facility: Azienda Ospedale-Università di Padova
city: Pisa
country: Italy
facility: Azienda Ospedaliero Universitaria Pisana (AOUP)
city: Rozzano
country: Italy
facility: Istituto Clinico Humanitas
city: Ōmura
country: Japan
facility: National Hospital Organization Nagasaki Medical Center
state: Nagasaki
city: Ehime
country: Japan
facility: Ehime University Hospital
city: Tokyo
country: Japan
facility: Toranomon Hospital
city: Tokyo
country: Japan
facility: Juntendo University Hospital
city: Amsterdam
country: Netherlands
facility: Amstedam University Medical Centers
city: Nijmegen
country: Netherlands
facility: Radboudumc
city: Rotterdam
country: Netherlands
facility: Erasmus medical center
city: Barcelona
country: Spain
facility: Hospital Clínic de Barcelona
city: Barcelona
country: Spain
facility: Hospital Universitari Parc Tauli
city: Madrid
country: Spain
facility: Hospital General Universitario Gregorio Maranon
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocio
city: Southampton
country: United Kingdom
facility: University Hospital Southampton NHS Foundation Trust
state: Hampshire
city: Belfast
country: United Kingdom
facility: Belfast Health & Social Care Trust (BHSCT) Royal Victoria Hospital
city: London
country: United Kingdom
facility: King's College Hospital NHS Foundation Trust
city: Nottingham
country: United Kingdom
facility: Nottingham University Hospitals NHS Trust
Study officials:
References: