NCT04663308

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

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

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 182 (actual)
• Sponsor: Mirum Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Primary Sclerosing Cholangitis

Interventions

Type	Name	Description
DRUG	Volixibat	Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.
DRUG	Placebo	Capsules matched to study drug minus active substance

Timeline

• Start date: 2020-12-18
• Primary completion: 2026-06-01
• Completion: 2027-04-01
• First posted: 2020-12-11
• Last updated: 2026-02-04

Locations

103 sites across 14 countries: United States, Argentina, Australia, Belgium, Brazil, Canada, France, Germany, Israel, Italy, Netherlands, Spain, Switzerland, United Kingdom

Regulatory

• FDA-regulated drug study