NCT03905330

A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)

MRX-502: Randomized Double-blind Placebo-controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC) - MARCH-PFIC

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 93 (actual)
• Sponsor: Mirum Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 1 Year – 17 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to determine whether the investigational treatment (maralixibat) is safe and effective in pediatric participants with Progressive Familial Intrahepatic Cholestasis (PFIC).

Detailed description

This study was conducted at multiple sites in North America, Europe, Asia and South America.

Conditions

• Progressive Familial Intrahepatic Cholestasis (PFIC)

Interventions

Type	Name	Description
DRUG	Maralixibat	Maralixibat oral solution (up to 600 mcg/kg) orally twice daily for 26 weeks.
OTHER	Placebo	Placebo matching to maralixibat orally twice daily for 26 weeks.

Timeline

• Start date: 2019-07-09
• Primary completion: 2022-09-01
• Completion: 2022-09-01
• First posted: 2019-04-05
• Last updated: 2023-12-11
• Results posted: 2023-12-11

Locations

31 sites across 17 countries: United States, Argentina, Austria, Belgium, Brazil, Canada, Colombia, France, Germany, Hungary, Italy, Lebanon, Mexico, Poland, Singapore, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study