NCT05050136

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

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

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.

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; PBC patients with AIH are not excluded
* History of Liver transplantation

Disease(s) and\or Condition(s)

Primary Biliary Cholangitis

PBC

Primary Purpose
  • TREATMENT
Intervention/Treatment
    • Type: DRUG
    • Name: Volixibat
    • Description: Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.
    • Arm Group Labels: Volixibat 20mg
    • Type: DRUG
    • Name: Placebo
    • Description: Capsules matched to study drug without the active pharmaceutical ingredient
    • Arm Group Labels: Placebo
Sponsor
  • Mirum Pharmaceuticals, Inc.