NCT02057718

Open Label Study to Evaluate Efficacy and Long Term Safety of LUM001 (Maralixibat) in the Treatment of Cholestatic Liver Disease in Patients With Progressive Familial Intrahepatic Cholestasis

Open Label Study of the Efficacy and Long Term Safety of LUM001 (Maralixibat), an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Pediatric Patients With Progressive Familial Intrahepatic Cholestasis

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 33 (actual)
• Sponsor: Mirum Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 12 Months – 18 Years
• Healthy volunteers: Not accepted

Summary

This is an open label study in children with Progressive Familial Intrahepatic Cholestasis (PFIC) designed to evaluate the safety and efficacy of LUM001, also known as Maralixibat (MRX). Efficacy will be assessed by evaluating the effect of LUM001 on pruritus and the biochemical markers of pruritus associated with PFIC.

Detailed description

The study is divided into 5 parts: a 4-week dose escalation period, a 4-week stable dosing period, a 5-week stable dosing period, a 59-week long-term exposure period, and an optional follow-up treatment period for eligible participants who continue treatment with LUM001. Participants in the optional follow-up treatment period will continue to receive study drug until they are eligible to enter another LUM001 study or until LUM001 is available commercially, whichever occurs first.

Conditions

• Progressive Familial Intrahepatic Cholestasis (PFIC)

Interventions

Type	Name	Description
DRUG	LUM001 (Maralixibat)	LUM001 also known as Maralixibat (MRX) oral dose up to twice a day (BID).

Timeline

• Start date: 2014-03-01
• Primary completion: 2020-05-08
• Completion: 2020-05-08
• First posted: 2014-02-07
• Last updated: 2023-10-23
• Results posted: 2021-07-12

Locations

10 sites across 4 countries: United States, France, Poland, United Kingdom

Regulatory

• FDA-regulated drug study