A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC)
- Registration Number
- NCT04663308
- 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 Sclerosing Cholangitis (PSC), and to assess the possible impact on the disease progression of PSC.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 200
- 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.
- Pruritus associated with an etiology other than PSC
- Evidence or clinical suspicion of decompensated cirrhosis, or a history of decompensation events
- History of ileostomy or small bowel surgery/resection or other surgeries that may have disrupted the enterohepatic circulation
- Evidence, history, or suspicion of other liver disease; PSC patients with AIH are not excluded.
- Bile duct stent or percutaneous bile duct drain placement, or balloon dilatation procedure of a stricture within 12 weeks of Screening
- Exceeding pre-defined biochemical values for alanine aminotransferase/aspartate aminotransferase (ALT/AST), estimated glomerular filtration rate (eGFR),serum creatinine (sCr), platelet count, international normalized ratio (INR) and total bilirubin
- 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 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 2: Placebo Placebo Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily. Part 1 Arm 2: Volixibat 80mg Volixibat Participants randomized to this arm will receive volixibat 80mg twice daily. Part 2 Arm 1: Volixibat Selected Dose 20mg Volixibat Participants randomized to this arm will receive volixibat 20mg 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 through 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 The incidence of adverse events Baseline through to Week 28 Proportion of participants with itch response using the Adult ItchRO Baseline through to Week 28 Changes in serum bile acid levels Baseline through to Week 28 Changes in alkaline phosphatase Baseline through to Week 28 Changes in total bilirubin levels Baseline through to Week 28 Change in Primary Sclerosing Cholangitis-Specific Patient-Reported Outcome Baseline through to Week 28 Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) fatigue questionnaire Baseline through 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 through 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 (103)
McMaster University Hospital
🇨🇦Hamilton, Ontario, Canada
Southern California Research Center
🇺🇸Coronado, California, United States
Cedars Sinai Medical Center
🇺🇸Los Angeles, California, United States
University of California, Davis
🇺🇸Sacramento, California, United States
California Pacific Medical Center Research Institute
🇺🇸San Francisco, California, United States
University of Colorado - Anschutz Medical Campus
🇺🇸Aurora, Colorado, United States
MedStar Georgetown University Hospital
🇺🇸Washington, District of Columbia, United States
Florida Research Institute
🇺🇸Lakewood Ranch, Florida, United States
University of Miami - Schiff Center for Liver Diseases
🇺🇸Miami, Florida, United States
Tampa General Hospital
🇺🇸Tampa, Florida, United States
Beth Israel Deaconess Medical Center
🇺🇸Boston, Massachusetts, United States
Henry Ford Hospital
🇺🇸Detroit, Michigan, United States
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
Southern Therapy and Advanced Research LLC
🇺🇸Jackson, Mississippi, United States
Northwell Health
🇺🇸Manhasset, New York, United States
New York University Langone Health
🇺🇸New York, New York, United States
Weill Cornell Medical College
🇺🇸New York, New York, United States
Columbia University Medical Center - Presbyterian Hospital and Vanderbilt Clinic
🇺🇸New York, New York, United States
University of Rochester Medical Center - Strong Memorial Hospital
🇺🇸Rochester, New York, United States
Duke Health - Duke University Medical Center
🇺🇸Durham, North Carolina, United States
Science 37, Inc (Remote-homebased Telemedicine)
🇺🇸Morrisville, North Carolina, United States
Cleveland Clinic
🇺🇸Cleveland, Ohio, United States
Einstein Healthcare Network - Einstein Medical Center
🇺🇸Philadelphia, Pennsylvania, United States
University of Pittsburgh Medical Center
🇺🇸Pittsburgh, Pennsylvania, United States
Galen Medical Group
🇺🇸Hixson, Tennessee, United States
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
The Liver Institute At Methodist Dallas Medical Center
🇺🇸Dallas, Texas, United States
University of Texas Southwestern Medical Center
🇺🇸Dallas, Texas, United States
Houston Methodist Hospital
🇺🇸Houston, Texas, United States
Liver Associates of Texas
🇺🇸Houston, Texas, United States
University of Utah Health Care
🇺🇸Salt Lake City, Utah, United States
Bon Secours Liver Institution of Hampton Roads Mary Immaculate Hospital Office
🇺🇸Newport News, Virginia, United States
Richmond Community Hospital LLC
🇺🇸Richmond, Virginia, United States
University of Washington - Harborview Medical Center
🇺🇸Seattle, Washington, United States
Liver Institute Northwest
🇺🇸Seattle, Washington, United States
Hospital Italiano de Buenos Aires
🇦🇷Buenos Aires, Argentina
Glenny Corp S.A.
🇦🇷Ciudad Autónoma Buenos Aires, Argentina
Hospital El Cruce
🇦🇷Florencio Varela, Argentina
Royal Adelaide Hospital
🇦🇺Adelaide, Australia
Northern Health
🇦🇺Epping, Australia
Liverpool Hospital
🇦🇺Liverpool, Australia
Austin Health
🇦🇺Melbourne, Australia
University of the Sunshine Coast
🇦🇺Sippy Downs, Australia
Cliniques Universitaires Saint-Luc
🇧🇪Brussels, Belgium
Universitair Ziekenhuis Antwerpen (UZA)
🇧🇪Gent, Belgium
University Hospital Ghent
🇧🇪Gent, Belgium
Hospital das Clinicas da UFMG
🇧🇷Belo Horizonte, Brazil
Hospital Das Clínicas Da Ufg
🇧🇷Goiânia, Brazil
Hospital de Clinicas de Porto Alegre
🇧🇷Porto Alegre, Brazil
Escola Bahiana de Medicina e Saude Publica
🇧🇷Salvador, Brazil
Hospital do Rim e Hipertensao
🇧🇷São Paulo, Brazil
University of Alberta
🇨🇦Edmonton, Alberta, Canada
The Ottawa Hospital - General Campus
🇨🇦Ottawa, Ontario, Canada
University Health Network - Toronto General Hospital
🇨🇦Toronto, Ontario, Canada
University of Calgary
🇨🇦Calgary, Canada
London Health Sciences Centre University Hospital
🇨🇦London, Canada
McGill University Health Centre - Royal Victoria Hospital
🇨🇦Montréal, Canada
Centre Hospitalier Universitaire Grenoble Alpes (CHU Grenoble Alpes)
🇫🇷Grenoble, France
Centre Hospitalier Universitaire de Lille
🇫🇷Lille Cedex, France
APHP-Hôpital Saint Antoine
🇫🇷Paris Cedex 12, France
CHU Bordeaux - Haut Lévêque
🇫🇷Pessac, France
Hannover Medical School
🇩🇪Hannover, Lower Saxony, Germany
Universitaetsklinikum Bonn
🇩🇪Bonn, Nordrhine-Westphalia, Germany
Charité Universtitätsmedizin - Campus Virchow-Klinikum
🇩🇪Berlin, Germany
Universitaetsklinikum Erlangen
🇩🇪Erlangen, Germany
Universitätsklinikum Schleswig-Holstein - Campus Kiel
🇩🇪Kiel, Germany
Universitatsklinikum Leipzig
🇩🇪Leipzig, Germany
Otto-von-Guericke-Universitat Magdeburg
🇩🇪Magdeburg, Germany
University Hospital Tubingen 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
🇮🇱Jerusalem, Israel
Galilee Medical Center
🇮🇱Nahariyya, Israel
Rabin Medical Center
🇮🇱Petach Tikva, Israel
Sheba Medical Center
🇮🇱Ramat Gan, Israel
Tel Aviv Sourasky Medical Center
🇮🇱Tel Aviv, Israel
Azienda Ospedaliero-Universitaria di Bologna - Policlinico S. Orsola-Malpighi
🇮🇹Bologna, Italy
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
🇮🇹Milan, Italy
ASST Grande Ospedale Metropolitano Niguarda
🇮🇹Milan, Italy
Fondazione IRCCS San Gerardo dei Tintori
🇮🇹Monza, Italy
A.O.U. Federico II
🇮🇹Napoli, Italy
Azienda Ospedale Universita Padova
🇮🇹Padova, Italy
Amsterdam UMC - VU Medisch Centrum (VUmc)
🇳🇱Amsterdam, Netherlands
Universitair Medisch Centrum Groningen
🇳🇱Groningen, Netherlands
Radboud University Medical Centre (MC)
🇳🇱Nijmegen, Netherlands
Erasmus University Medical Center
🇳🇱Rotterdam, Netherlands
Hospital Clinic Barcelona
🇪🇸Barcelona, Spain
Hospital General Universitario Gregorio Maranon
🇪🇸Madrid, Spain
University Hospital Zurich
🇨🇭Zürich, Switzerland
Royal Free Hospital
🇬🇧London, Hampstead, United Kingdom
John Radcliffe Hospital
🇬🇧Oxford, Headington, United Kingdom
Royal Victoria Hospital
🇬🇧Belfast, United Kingdom
University Hospitals Birmingham NHS Foundation Trust - Wellcome Trust Birmingham Clinical Research Facility
🇬🇧Birmingham, United Kingdom
Cambridge University Hospitals NHS Foundation Trust - Addenbrooke's Hospital
🇬🇧Cambridge, United Kingdom
Lothian NHS Board
🇬🇧Edinburgh, United Kingdom
St James' University Hospital Leeds Teaching Hospitals NHS Trust
🇬🇧Leeds, United Kingdom
King's College Hospital NHS Foundation Trust
🇬🇧London, United Kingdom
The Newcastle upon Tyne Hospitals NHS Foundation Trust - Royal Victoria Infirmary
🇬🇧Newcastle Upon Tyne, United Kingdom
Nottingham University Hospital NHS Trust
🇬🇧Nottingham, United Kingdom
University Hospital Southampton NHS Foundation Trust
🇬🇧Southampton, United Kingdom