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

Phase 2RecruitingNCT05050136

Mirum Pharmaceuticals, Inc.260 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

260

Conditions

Primary Biliary Cholangitis PBC

Interventions

VolixibatDRUG

Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.

PlaceboDRUG

Capsules matched to study drug without the active pharmaceutical ingredient

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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, anti-pruritic medication and PPAR agonist 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; PBC patients with AIH are not excluded * History of Liver transplantation

Locations (115)

Outcomes

Primary Outcomes

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.