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

Part of paid clinical trials in Coronado, California.

Sponsor
Mirum Pharmaceuticals, Inc.
Study ID
NCT05050136
Phase
PHASE2
Status
Recruiting

Conditions

  • PBC
  • Primary Biliary Cholangitis

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

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

Study Details

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.

Key Dates

Start date
Sep 22, 2021
Status verified
May 2026
Primary completion
Apr 30, 2027
Completion
Jul 31, 2027

Study Design

Enrollment
260 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Part 1 Arm 1: Volixibat 20mg
    Participants randomized to this arm will receive volixibat 20mg twice daily.
  • Experimental: Part 1 Arm 2: Volixibat 80mg
    Participants randomized to this arm will receive volixibat 80mg twice daily.
  • Placebo Comparator: Part 1 Arm 3: Placebo
    Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
  • Experimental: Part 2 Arm 1: Volixibat Selected Dose 20mg
    Participants randomized to this arm will receive volixibat 20mg twice daily.
  • Placebo Comparator: Part 2 Arm 2: Placebo
    Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.

Primary Outcome Measure

Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire [ Time Frame: Baseline to week 28 ]

Locations (44)

FacilityCityStateZIPSite coordinators
Southern California Research CenterCoronadoCalifornia92218-
Cedars Sinai Medical CenterLos AngelesCalifornia90048
Nirosha Tilakaratna
310-423-1269
Kevin Yashar
310-423-2743
University of California Los AngelesLos AngelesCalifornia90095
Marco Morcos
310-825-9225
UC DavisSacramentoCalifornia95817
Sandeep Dhaliwal
916-734-8696
University of ColoradoAuroraColorado80045
Charles Stasa
303-724-6875
Yale University Digestive DiseasesNew HavenConnecticut06510
Suzie Christopher
203-785-4070
Covenant Metabolic SpecialistsFort MyersFlorida33912-
UF Hepatology Research at CTRBGainesvilleFlorida32610
Lasheaka McClellan
352-273-7285
UF Health Gastroenterology- EmersonJacksonvilleFlorida32209-
IHS HealthKissimmeeFlorida34741-
Schiff Center for Liver DiseasesMiamiFlorida33136-
Advanced Research Institute, IncNew Port RicheyFlorida34653
Christina Tyson
727-835-3261
Covenant Research and Clinics, LLCSarasotaFlorida34240-
Tampa General HospitalTampaFlorida33606
Jennifer Schmidt, RN, BSN
813-347-2878
Cleveland Clinic FloridaWestonFlorida33331
Juan Armijos
954-659-6215
Southeast Clinical Research CenterDaltonGeorgia30720-
Northwestern UniversityChicagoIllinois60611-
Loyola University Health System - Loyola Outpatient CenterMaywoodIllinois60153
Vira Boichuk
708-216-3268
Michelle Kudulis
708-216-0291
University of Iowa Hospitals & ClinicsIowa CityIowa52242
Jena Valestin, RN, BSN
319-353-4574
Ochsner Clinic Foundation-Baton RougeBaton RougeLouisiana70836
Magen Parker
225-791-5278
LSU Health Science Center ShreveportShreveportLouisiana71103
Robyne Champagne
318-675-5803
Beth Israel DeaconessBostonMassachusetts02215
Julie Shea
617-632-1125
Massachusetts General HospitalBostonMassachusetts02114
Daniel Pratt
617-726-2426
Mayo ClinicRochesterMinnesota55905
Mitchell Clayton
507-284-2698
Jackson Liver and GI SpecialistsJacksonMississippi39216-
CHI Health Clinic (Alegent Creighton Clinic)- Gastroenterology-BerganOmahaNebraska68124
Madeline Esch
402-343-8511
Virtua Center for Liver Disease-CamdenCamdenNew Jersey08103
Donna Hoopes
856-355-1206
Montefiore Medical CenterThe BronxNew York10467
Paola Mejia
347-671-8217
Science 37, Inc (Remote-homebased Telemedicine)MorrisvilleNorth Carolina27560
Clinical Research Coordinator
888-462-7559
Cleveland Clinic - Main CampusClevelandOhio44195
Kevin Kruszewski
216-636-5555
Erin McMullen
216-636-5555
Einstein Healthcare Network - Einstein Medical CenterPhiladelphiaPennsylvania19141
Laura Clark
215-456-8721
Methodist Le Bonheur HealthcareMemphisTennessee38104-
Galen Medical Group, P.CTennessee CityTennessee37343
Kim Norwood
423-954-9015
Sydney Boles
423-954-9015
Amel Med LLCAustinTexas78750-
The Liver Institute at Methodist Dallas Medical CenterDallasTexas75203
Colette Ngo Ndjom
214-947-1289
Soma Clinical TrialsDenisonTexas75020-
Baylor College of MedicineHoustonTexas77030
Youseph Karouni
713-798-3706
Liver Associates of TexasHoustonTexas77030
Quynh-An Phan
713-799-2667
Impact ResearchWacoTexas76710-
University of Utah Health CareSalt Lake CityUtah84132
Amy Holman
801-538-0689
MedStar Health Research InstituteFairfaxVirginia22031
Grace Jin
202-877-6501
Bon Secours Liver Institute of Hampton Roads Mary Immaculate HospitalNewport NewsVirginia23602-
Bon Secours Richmond Community Hospital LLC d/b/a Bon Secours Liver Institute of RichmondRichmondVirginia23226
Laura Joines
804-977-8921
Liver Institute NorthwestSeattleWashington98105
Theresa Dorrian
206-536-3030

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