A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC)

Primary Sclerosing Cholangitis Clinical Trial

— VISTAS  

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

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
• 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
• 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

Related Conditions & MeSH terms

• Cholangitis
• Cholangitis, Sclerosing
• Primary Sclerosing Cholangitis

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 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.	Baseline through to Week 28
Secondary	Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) sleep disturbance questionnaire	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.	Baseline through to Week 28