NCT04168385

MRX-800: A Long-Term Safety Study of Maralixibat in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study

MRX-800: A Long-Term Safety Study of Maralixibat, an Apical Sodium Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 52 (actual)
• Sponsor: Mirum Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 12 Months
• Healthy volunteers: Not accepted

Summary

Evaluate the long-term safety of maralixibat (MRX) in subjects with cholestatic liver disease including, but not limited to, Alagille Syndrome (ALGS), Progressive Familial Intrahepatic Cholestasis (PFIC) and Biliary Atresia.

Detailed description

This is a multicenter, open-label study of maralixibat in subjects diagnosed with cholestatic liver disease (including, but not limited to ALGS, PFIC or Biliary Atresia) who have previously participated in a maralixibat clinical study. All subjects received maralixibat in the previous studies and will continues to receive maralixibat in this study.

Conditions

• Cholestatic Liver Disease

Interventions

Type	Name	Description
DRUG	Maralixibat	Maralixibat chloride oral solution orally twice daily (up to 1200\* mcg/kg/day), and according to indication. \*equivalent to 1140 mcg/kg/day maralixibat

Timeline

• Start date: 2020-01-16
• Primary completion: 2024-09-04
• Completion: 2024-09-04
• First posted: 2019-11-19
• Last updated: 2025-12-08
• Results posted: 2025-12-08

Locations

17 sites across 8 countries: United States, Australia, Belgium, Canada, France, Poland, Spain, United Kingdom

Regulatory

• FDA-regulated drug study