A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Biliary Cholangitis
- Registration Number
- NCT05050136
- Lead Sponsor
- Mirum Pharmaceuticals, Inc.
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 180
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Part 1 Arm 1: Volixibat 20mg Volixibat Participants randomized to this arm will receive volixibat 20mg twice daily. Part 1 Arm 2: Volixibat 80mg Volixibat Participants randomized to this arm will receive volixibat 80mg twice daily. Part 1 Arm 3: Placebo Placebo Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily. Part 2 Arm 1: Volixibat Selected Dose 20mg Volixibat Participants randomized to this arm will receive volixibat 20mg twice daily. Part 2 Arm 2: Placebo Placebo Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
- Primary Outcome Measures
Name Time Method Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire Baseline 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.
- Secondary Outcome Measures
Name Time Method Incidence of adverse events Baseline to week 28 Changes in alkaline phosphatase Baseline to week 28 Changes in total bilirubin Baseline to week 28 Proportion of participants with itch response using the Adult ItchRO Baseline to week 28 Changes in serum bile acid levels Baseline to week 28 Change in Primary Biliary Cholangitis Quality of Life Measure (PBC-40) Baseline to week 28 PBC-40 questionnaire is scored on a scale of 40 to 200, with higher scores indicating poorer quality of life
Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) fatigue questionnaire Baseline 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.
Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) sleep disturbance questionnaire Baseline 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.
Trial Locations
- Locations (91)
Southern California Research Center
🇺🇸Coronado, California, United States
Cedars Sinai Medical Center
🇺🇸Los Angeles, California, United States
University of Colorado
🇺🇸Aurora, Colorado, 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
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
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
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, Tennessee, United States
Amel Med LLC
🇺🇸Austin, Texas, United States
Soma Clinical Trials
🇺🇸Denison, Texas, United States
Liver Associates of Texas
🇺🇸Houston, Texas, United States
Velocity Clinical 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
🇦🇷Florencio Varela, Buenos Aires, Argentina
Glenny Corp S.A
🇦🇷Caba, Argentina
Universitair Ziekenhuis Antwerpe UZA
🇧🇪Antwerp, Belgium
Cliniques Universitaires Saint-Luc
🇧🇪Brussel, Belgium
UZ Gent
🇧🇪Gent, Belgium
Centro de Pesquisa Clínica da Bahiana Saúde
🇧🇷Salvador, Bahia, Brazil
Centro de Pesquisas Clínicas do HC/UFMG-EBSERH
🇧🇷Belo Horizonte, Minas Gerais, Brazil
Hospital de Clínicas de Porto Alegre
🇧🇷Porto Alegre, RS, Brazil
Fundação Oswaldo Ramos
🇧🇷São Paulo, Brazil
Gordon and Leslie Diamond Health Care Centre, Division of Gastroenterology
🇨🇦Vancouver, British Columbia, Canada
University Hospital, LHSC
🇨🇦London, Ontario, Canada
University of Alberta
🇨🇦Edmonton, Canada
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
University Hospital Munster
🇩🇪Münster, Germany
University Hospital Tübingen Medical clinic
🇩🇪Tubingen, 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
🇮🇱Nahariyya, Israel
Rabin Medical Center
🇮🇱Petach 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
Fondazione Ca Granda
🇮🇹Milano, Italy
ASST Grande Ospedale Metropolitano Niguarda
🇮🇹Milano, Italy
Fondazione IRCCS San Gerardo dei Tintori
🇮🇹Monza, Italy
Azienda Ospedale-Università di Padova
🇮🇹Padova, 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
Toranomon Hospital
🇯🇵Tokyo, Japan
Juntendo University Hospital
🇯🇵Tokyo, Japan
Amstedam University Medical Centers
🇳🇱Amsterdam, Netherlands
Radboudumc
🇳🇱Nijmegen, Netherlands
Erasmus medical center
🇳🇱Rotterdam, Netherlands
Hospital Clínic de Barcelona
🇪🇸Barcelona, Spain
Hospital Universitari Parc Tauli
🇪🇸Barcelona, Spain
Hospital General Universitario Gregorio Maranon
🇪🇸Madrid, Spain
Hospital Universitario Virgen del Rocio
🇪🇸Sevilla, Spain
University Hospital Southampton NHS Foundation Trust
🇬🇧Southampton, Hampshire, United Kingdom
Belfast Health & Social Care Trust (BHSCT) Royal Victoria Hospital
🇬🇧Belfast, United Kingdom
King's College Hospital NHS Foundation Trust
🇬🇧London, United Kingdom
Nottingham University Hospitals NHS Trust
🇬🇧Nottingham, United Kingdom