Clinical trial NCT04663308: A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC)
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 Sclerosing Cholangitis
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Primary Sclerosing Cholangitis
Keywords: Pruritus
PSC
Itch
Itching
Cholestasis
Lead sponsor: Mirum Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2020-12-11
Enrollment: 200
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
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 Sclerosing Cholangitis (PSC), and to assess the possible impact on the disease progression of PSC.


Eligibility criteria: Inclusion Criteria:

1. 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.
2. Subjects aged ≥12 years for eligible regions; otherwise ≥18 years
3. Confirmed diagnosis of large duct or small duct PSC based on American Association for the Study of Liver Disease (AASLD) guidelines.
4. Pruritus associated with PSC as assessed by Adult ItchRO.
5. Ursodeoxycholic acid (UDCA) and anti-pruritic medication use will be allowed if meeting additional criteria.
6. Concomitant Inflammatory Bowel Disease (IBD) is allowed if meeting additional criteria.

Exclusion Criteria:

1. Pruritus associated with an etiology other than PSC
2. Evidence or clinical suspicion of decompensated cirrhosis, or a history of decompensation events
3. History of ileostomy or small bowel surgery/resection or other surgeries that may have disrupted the enterohepatic circulation
4. Evidence, history, or suspicion of other liver disease; PSC patients with AIH are not excluded.
5. Bile duct stent or percutaneous bile duct drain placement, or balloon dilatation procedure of a stricture within 12 weeks of Screening
6. 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
7. 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 through to Week 28
Secondary outcomes:
measure: Proportion of participants with itch response using the Adult ItchRO
timeFrame: Baseline through to Week 28
measure: The incidence of adverse events
timeFrame: Baseline through to Week 28
measure: Changes in serum bile acid levels
timeFrame: Baseline through to Week 28
measure: Changes in alkaline phosphatase
timeFrame: Baseline through to Week 28
measure: Changes in total bilirubin levels
timeFrame: Baseline through to Week 28
measure: Change in Primary Sclerosing Cholangitis-Specific Patient-Reported Outcome
timeFrame: Baseline through to Week 28
measure: Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) fatigue questionnaire
timeFrame: Baseline through to Week 28
measure: Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) sleep disturbance questionnaire
timeFrame: Baseline through 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: Sacramento
country: United States
facility: University of California, Davis
state: California
city: San Francisco
country: United States
facility: California Pacific Medical Center Research Institute
state: California
city: Aurora
country: United States
facility: University of Colorado - Anschutz Medical Campus
state: Colorado
city: Washington
country: United States
facility: MedStar Georgetown University Hospital
state: District of Columbia
city: Lakewood Ranch
country: United States
facility: Florida Research Institute
state: Florida
city: Miami
country: United States
facility: University of Miami - Schiff Center for Liver Diseases
state: Florida
city: Tampa
country: United States
facility: Tampa General Hospital
state: Florida
city: Boston
country: United States
facility: Beth Israel Deaconess Medical Center
state: Massachusetts
city: Detroit
country: United States
facility: Henry Ford Hospital
state: Michigan
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Jackson
country: United States
facility: Southern Therapy and Advanced Research LLC
state: Mississippi
city: Manhasset
country: United States
facility: Northwell Health
state: New York
city: New York
country: United States
facility: New York University Langone Health
state: New York
city: New York
country: United States
facility: Weill Cornell Medical College
state: New York
city: New York
country: United States
facility: Columbia University Medical Center - Presbyterian Hospital and Vanderbilt Clinic
state: New York
city: Rochester
country: United States
facility: University of Rochester Medical Center - Strong Memorial Hospital
state: New York
city: Durham
country: United States
facility: Duke Health - Duke University Medical Center
state: North Carolina
city: Morrisville
country: United States
facility: Science 37, Inc (Remote-homebased Telemedicine)
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Philadelphia
country: United States
facility: Einstein Healthcare Network - Einstein Medical Center
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center
state: Pennsylvania
city: Hixson
country: United States
facility: Galen Medical Group
state: Tennessee
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Dallas
country: United States
facility: The Liver Institute At Methodist Dallas Medical Center
state: Texas
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center
state: Texas
city: Houston
country: United States
facility: Houston Methodist Hospital
state: Texas
city: Houston
country: United States
facility: Liver Associates of Texas
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah Health Care
state: Utah
city: Newport News
country: United States
facility: Bon Secours Liver Institution of Hampton Roads Mary Immaculate Hospital Office
state: Virginia
city: Richmond
country: United States
facility: Richmond Community Hospital LLC
state: Virginia
city: Seattle
country: United States
facility: University of Washington - Harborview Medical Center
state: Washington
city: Seattle
country: United States
facility: Liver Institute Northwest
state: Washington
city: Buenos Aires
country: Argentina
facility: Hospital Italiano de Buenos Aires
city: Ciudad Autónoma Buenos Aires
country: Argentina
facility: Glenny Corp S.A.
city: Florencio Varela
country: Argentina
facility: Hospital El Cruce
city: Adelaide
country: Australia
facility: Royal Adelaide Hospital
city: Epping
country: Australia
facility: Northern Health
city: Liverpool
country: Australia
facility: Liverpool Hospital
city: Melbourne
country: Australia
facility: Austin Health
city: Sippy Downs
country: Australia
facility: University of the Sunshine Coast
city: Brussels
country: Belgium
facility: Cliniques Universitaires Saint-Luc
city: Gent
country: Belgium
facility: Universitair Ziekenhuis Antwerpen (UZA)
city: Gent
country: Belgium
facility: University Hospital Ghent
city: Belo Horizonte
country: Brazil
facility: Hospital das Clinicas da UFMG
city: Goiânia
country: Brazil
facility: Hospital Das Clínicas Da Ufg
city: Porto Alegre
country: Brazil
facility: Hospital de Clinicas de Porto Alegre
city: Salvador
country: Brazil
facility: Escola Bahiana de Medicina e Saude Publica
city: São Paulo
country: Brazil
facility: Hospital do Rim e Hipertensao
city: Edmonton
country: Canada
facility: University of Alberta
state: Alberta
city: Hamilton
country: Canada
facility: McMaster University Hospital
state: Ontario
city: Ottawa
country: Canada
facility: The Ottawa Hospital - General Campus
state: Ontario
city: Toronto
country: Canada
facility: University Health Network - Toronto General Hospital
state: Ontario
city: Calgary
country: Canada
facility: University of Calgary
city: London
country: Canada
facility: London Health Sciences Centre University Hospital
city: Montréal
country: Canada
facility: McGill University Health Centre - Royal Victoria Hospital
city: Grenoble
country: France
facility: Centre Hospitalier Universitaire Grenoble Alpes (CHU Grenoble Alpes)
city: Lille Cedex
country: France
facility: Centre Hospitalier Universitaire de Lille
city: Paris Cedex 12
country: France
facility: APHP-Hôpital Saint Antoine
city: Pessac
country: France
facility: CHU Bordeaux - Haut Lévêque
city: Hannover
country: Germany
facility: Hannover Medical School
state: Lower Saxony
city: Bonn
country: Germany
facility: Universitaetsklinikum Bonn
state: Nordrhine-Westphalia
city: Berlin
country: Germany
facility: Charité Universtitätsmedizin - Campus Virchow-Klinikum
city: Erlangen
country: Germany
facility: Universitaetsklinikum Erlangen
city: Kiel
country: Germany
facility: Universitätsklinikum Schleswig-Holstein - Campus Kiel
city: Leipzig
country: Germany
facility: Universitatsklinikum Leipzig
city: Magdeburg
country: Germany
facility: Otto-von-Guericke-Universitat Magdeburg
city: Tübingen
country: Germany
facility: University Hospital Tubingen 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
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: Tel Aviv Sourasky Medical Center
city: Bologna
country: Italy
facility: Azienda Ospedaliero-Universitaria di Bologna - Policlinico S. Orsola-Malpighi
city: Milan
country: Italy
facility: Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
city: Milan
country: Italy
facility: ASST Grande Ospedale Metropolitano Niguarda
city: Monza
country: Italy
facility: Fondazione IRCCS San Gerardo dei Tintori
city: Napoli
country: Italy
facility: A.O.U. Federico II
city: Padova
country: Italy
facility: Azienda Ospedale Universita Padova
city: Amsterdam
country: Netherlands
facility: Amsterdam UMC - VU Medisch Centrum (VUmc)
city: Groningen
country: Netherlands
facility: Universitair Medisch Centrum Groningen
city: Nijmegen
country: Netherlands
facility: Radboud University Medical Centre (MC)
city: Rotterdam
country: Netherlands
facility: Erasmus University Medical Center
city: Barcelona
country: Spain
facility: Hospital Clinic Barcelona
city: Madrid
country: Spain
facility: Hospital General Universitario Gregorio Maranon
city: Zürich
country: Switzerland
facility: University Hospital Zurich
city: London
country: United Kingdom
facility: Royal Free Hospital
state: Hampstead
city: Oxford
country: United Kingdom
facility: John Radcliffe Hospital
state: Headington
city: Belfast
country: United Kingdom
facility: Royal Victoria Hospital
city: Birmingham
country: United Kingdom
facility: University Hospitals Birmingham NHS Foundation Trust - Wellcome Trust Birmingham Clinical Research Facility
city: Cambridge
country: United Kingdom
facility: Cambridge University Hospitals NHS Foundation Trust - Addenbrooke's Hospital
city: Edinburgh
country: United Kingdom
facility: Lothian NHS Board
city: Leeds
country: United Kingdom
facility: St James' University Hospital Leeds Teaching Hospitals NHS Trust
city: London
country: United Kingdom
facility: King's College Hospital NHS Foundation Trust
city: Newcastle Upon Tyne
country: United Kingdom
facility: The Newcastle upon Tyne Hospitals NHS Foundation Trust - Royal Victoria Infirmary
city: Nottingham
country: United Kingdom
facility: Nottingham University Hospital NHS Trust
city: Southampton
country: United Kingdom
facility: University Hospital Southampton NHS Foundation Trust
Study officials:
References: