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

Primary Biliary Cholangitis Clinical Trial

— VANTAGE  

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 Biliary Cholangitis

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05050136
• Other study ID #: VLX-601
• Status: Recruiting
• Phase: Phase 2
• Start date: September 22, 2021
• Est. completion date: December 2025

Study information

• Verified date: April 2024
• Source: Mirum Pharmaceuticals, Inc.
• Contact: n/a
• 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 Biliary Cholangitis (PBC), and to assess the possible impact on the disease progression of PBC.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 180
• Est. completion date: December 2025
• Est. primary completion date: December 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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

Related Conditions & MeSH terms

• Cholangitis
• Liver Cirrhosis, Biliary
• PBC
• Primary Biliary Cholangitis

Intervention

Drug:
• Volixibat
Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.
• Placebo
Capsules matched to study drug without the active pharmaceutical ingredient

Locations

Country	Name	City	State
France	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
Germany	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
Israel	Hillel Yaffe Medical Center	Hadera
Israel	Carmel Medical Center	Haifa
Israel	Rambam Medical Center	Haifa
Israel	Hadassah Medical Center - Ein Karem, Liver Institute Kiryat Hadassah POB 12000	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	The Tel Aviv Sourasky Medical Center	Tel Aviv
Italy	Universita Politecnica delle Marche- Ospedali Riuniti	Ancona
Italy	ASST Grande Ospedale Metropolitano Niguarda	Milano
Italy	Fondazione Ca Granda	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
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 States	University of Colorado	Aurora	Colorado
United States	Amel Med LLC	Austin	Texas
United States	Ochsner Clinic Foundation-Baton Rouge	Baton Rouge	Louisiana
United States	Beth Israel Deaconess	Boston	Massachusetts
United States	Northwestern University	Chicago	Illinois
United States	Cleveland Clinic - Main Campus	Cleveland	Ohio
United States	Southern California Research Center	Coronado	California
United States	Science 37, Inc (Remote-homebased Telemedicine)	Culver City	California
United States	Southeast Clinical Research Center	Dalton	Georgia
United States	Soma Clinical Trials	Denison	Texas
United States	MedStar Health Research Institute	Fairfax	Virginia
United States	Covenant Metabolic Specialists	Fort Myers	Florida
United States	UF Hepatology Research at CTRB	Gainesville	Florida
United States	Liver Associates of Texas	Houston	Texas
United States	University of Iowa Hospitals & Clinics	Iowa City	Iowa
United States	Jackson Liver and GI Specialists	Jackson	Mississippi
United States	UF Health Gastroenterology- Emerson	Jacksonville	Florida
United States	IHS Health	Kissimmee	Florida
United States	Cedars Sinai Medical Center	Los Angeles	California
United States	Loyola University Health System - Loyola Outpatient Center	Maywood	Illinois
United States	Methodist Le Bonheur Healthcare	Memphis	Tennessee
United States	Schiff Center for Liver Diseases	Miami	Florida
United States	Advanced Research Institute, Inc	New Port Richey	Florida
United States	Bon Secours Liver Institute of Hampton Roads Mary Immaculate Hospital	Newport News	Virginia
United States	CHI Health Clinic (Alegent Creighton Clinic)- Gastroenterology-Bergan	Omaha	Nebraska
United States	Einstein Healthcare Network - Einstein Medical Center	Philadelphia	Pennsylvania
United States	Bon Secours Richmond Community Hospital LLC d/b/a Bon Secours Liver Institute of Richmond	Richmond	Virginia
United States	Mayo Clinic	Rochester	Minnesota
United States	University of Utah Health Care	Salt Lake City	Utah
United States	Covenant Research and Clinics, LLC	Sarasota	Florida
United States	Liver Institute Northwest	Seattle	Washington
United States	LSU Health Science Center Shreveport	Shreveport	Louisiana
United States	Tampa General Hospital	Tampa	Florida
United States	Galen Medical Group, P.C	Tennessee	Tennessee
United States	Impact Research	Waco	Texas

Sponsors (1)

Lead Sponsor	Collaborator
Mirum Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  France,  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 to week 28
Secondary	Proportion of participants with itch response using the Adult ItchRO		Baseline to week 28
Secondary	Incidence of adverse events		Baseline to week 28
Secondary	Changes in alkaline phosphatase		Baseline to week 28
Secondary	Changes in total bilirubin		Baseline to week 28
Secondary	Changes in serum bile acid levels		Baseline to week 28
Secondary	Change in Primary Biliary Cholangitis Quality of Life Measure (PBC-40)	PBC-40 questionnaire is scored on a scale of 40 to 200, with higher scores indicating poorer quality of life	Baseline 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 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 to week 28