NCT04185363

An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)

An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 90 (actual)
• Sponsor: Mirum Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 1 Year – 18 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of this open label extension study is to evaluate the long-term safety and tolerability of maralixibat.

Detailed description

The study will be conducted at multiple sites in North America, Europe, Asia, and South America.

Conditions

• Progressive Familial Intrahepatic Cholestasis (PFIC)

Interventions

Type	Name	Description
DRUG	Maralixibat	All subjects will receive Maralixibat oral solution (up to 600 microgram per kilogram \[mcg/kg\]) twice daily

Timeline

• Start date: 2020-01-08
• Primary completion: 2025-04-23
• Completion: 2025-04-23
• First posted: 2019-12-04
• Last updated: 2025-06-29

Locations

28 sites across 16 countries: United States, Argentina, Austria, Belgium, Brazil, Canada, Colombia, France, Germany, Italy, Lebanon, Mexico, Poland, Singapore, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study