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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT04663308
Other study ID # VLX-301
Secondary ID 2020-003027-4120
Status Recruiting
Phase Phase 2
First received
Last updated
Start date December 18, 2020
Est. completion date December 2025

Study information

Verified date June 2024
Source Mirum Pharmaceuticals, Inc.
Contact Clinical Trials Mirum
Phone +16506674085
Email Clinicaltrials@mirumpharma.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

Status Recruiting
Enrollment 200
Est. completion date December 2025
Est. primary completion date September 2025
Accepts healthy volunteers No
Gender All
Age group 12 Years and older
Eligibility 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

Study Design


Intervention

Drug:
Volixibat
Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.
Placebo
Capsules matched to study drug minus active substance

Locations

Country Name City State
Belgium University Hospital Ghent Gent
Canada University of Alberta Edmonton Alberta
Canada The Ottawa Hospital - General Campus Ottawa Ontario
Canada University Health Network - Toronto General Hospital Toronto Ontario
Germany Charité Universtitätsmedizin - Campus Virchow-Klinikum Berlin
Germany Universitaetsklinikum Bonn Bonn Nordrhine-Westphalia
Germany Universitaetsklinikum Erlangen Erlangen
Germany Hannover Medical School Hannover Lower Saxony
Germany Universitätsklinikum Schleswig-Holstein - Campus Kiel Kiel
Germany Universitatsklinikum Leipzig Leipzig
Germany University Hospital Tubingen Medical Clinic Tübingen
Germany St. Josefs-Hospital Wiesbaden Wiesbaden
Israel Hillel Yaffe Medical Center Hadera
Israel Carmel Medical Center Haifa
Israel Rambam Medical Center Haifa
Israel Hadassah Medical Center - Ein Karem Jerusalem
Israel Shaare Zedek Medical Center 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
Italy Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan
Italy A.O.U. Federico II Napoli
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 Royal Free Hospital London Hampstead
United Kingdom Nottingham University Hospital NHS Trust Nottingham
United Kingdom John Radcliffe Hospital Oxford Headington
United Kingdom University Hospital Southampton NHS Foundation Trust Southampton
United States University of Colorado - Anschutz Medical Campus Aurora Colorado
United States Beth Israel Deaconess Medical Center Boston Massachusetts
United States Cleveland Clinic Cleveland Ohio
United States Southern California Research Center Coronado California
United States Science 37, Inc (Remote-homebased Telemedicine) Culver City California
United States The Liver Institute At Methodist Dallas Medical Center Dallas Texas
United States University of Texas Southwestern Medical Center Dallas Texas
United States Henry Ford Hospital Detroit Michigan
United States Duke Health - Duke University Medical Center Durham North Carolina
United States Galen Medical Group Hixson Tennessee
United States Houston Methodist Hospital Houston Texas
United States Liver Associates of Texas Houston Texas
United States Southern Therapy and Advanced Research LLC Jackson Mississippi
United States Florida Research Institute Lakewood Ranch Florida
United States Cedars Sinai Medical Center Los Angeles California
United States Northwell Health Manhasset New York
United States University of Miami - Schiff Center for Liver Diseases Miami Florida
United States Vanderbilt University Medical Center Nashville Tennessee
United States Columbia University Irving Medical Center - Research Pharmacy New York 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 Bon Secours Liver Institution of Hampton Roads Mary Immaculate Hospital Office Newport News Virginia
United States Einstein Healthcare Network - Einstein Medical Center Philadelphia Pennsylvania
United States University of Pittsburgh Medical Center Pittsburgh Pennsylvania
United States Richmond Community Hospital LLC Richmond Virginia
United States Mayo Clinic Rochester Minnesota
United States University of Rochester Medical Center - Strong Memorial Hospital Rochester New York
United States University of California, Davis Sacramento California
United States University of Utah Health Care Salt Lake City Utah
United States California Pacific Medical Center Research Institute San Francisco California
United States Liver Institute Northwest Seattle Washington
United States University of Washington - Harborview Medical Center Seattle Washington
United States Tampa General Hospital Tampa Florida
United States MedStar Georgetown University Hospital Washington District of Columbia

Sponsors (1)

Lead Sponsor Collaborator
Mirum Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Belgium,  Canada,  Germany,  Israel,  Italy,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire The Adult ItchRO is an 11-point NRS measurement of itch severity ranging from 0=no itch to 10=worst possible itch. Baseline through to Week 28
Secondary Proportion of participants with itch response using the Adult ItchRO Baseline through to Week 28
Secondary The incidence of adverse events Baseline through to Week 28
Secondary Changes in serum bile acid levels Baseline through to Week 28
Secondary Changes in alkaline phosphatase Baseline through to Week 28
Secondary Changes in total bilirubin levels Baseline through to Week 28
Secondary Change in Primary Sclerosing Cholangitis-Specific Patient-Reported Outcome Baseline through to Week 28
Secondary Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) fatigue questionnaire The PROMISĀ® Fatigue questionnaire is a 5-point response scale. Respondents endorse each item using a 5-point response scale: Never, Rarely, Sometimes, Often, and Always. Baseline through to Week 28
Secondary Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) sleep disturbance questionnaire The PROMISĀ® 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. Baseline through to Week 28
See also
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