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 — INDIGO  

Progressive Familial Intrahepatic Cholestasis (PFIC) Clinical Trial

Official title: 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

Clinical Trial 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.

Clinical Trial 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. ;

Related Conditions & MeSH terms

• Cholestasis
• Cholestasis, Intrahepatic
• Liver Diseases
• Progressive Familial Intrahepatic Cholestasis (PFIC)

• NCT number: NCT02057718
• Study type: Interventional
• Source: Mirum Pharmaceuticals, Inc.
• Status: Completed
• Phase: Phase 2
• Start date: March 1, 2014
• Completion date: May 8, 2020