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

Phase 2

Completed

• Conditions: Progressive Familial Intrahepatic Cholestasis (PFIC)
• Interventions: Drug: LUM001 (Maralixibat)

• Registration Number: NCT02057718
• Lead Sponsor: Mirum Pharmaceuticals, Inc.

Brief 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.

Study Timeline

• Study First Submitted: 2014-02-05
• Study First Submitted QC: 2014-02-05
• Study First Posted: 2014-02-07
• Study Start: 2014-03-01
• Primary Completion: 2020-05-08
• Study Completion: 2020-05-08
• Results First Submitted: 2021-03-17
• Results First Submitted QC: 2021-06-16
• Results First Posted: 2021-07-12
• Status Verified: 2023-10
• Last Update Submitted: 2023-10-18
• Last Update Posted: 2023-10-23

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 33

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
LUM001 (Maralixibat)	LUM001 (Maralixibat)	Participants will receive LUM001, also known as Maralixibat (MRX) twice a day (BID).

Primary Outcome Measures

Name	Time	Method
Change From Baseline to Endpoint (Week 13) in Fasting sBA Level	Baseline (Day 0) to Week 13

Secondary Outcome Measures

Name	Time	Method
Change From Baseline to Week 13/ET in Direct Bilirubin	Baseline (Day 0) to Week 13
Change From Baseline to Week 13/ET in Pruritus as Measured by ItchRO(Obs)	Baseline (Day 0) to Week 13	This secondary efficacy endpoint is the change from baseline to Week 13 in pruritus as measured by ItchRO(Obs) weekly average morning score. ItchRO scores range from 0 to 4; the higher score indicates increasing itch severity (0 = none; 4 = very severe).
Change From Baseline to Week 13/ET in Pruritus as Measured by ItchRO(Pt)	Baseline (Day 0) to Week 13	This secondary efficacy endpoint is the change from baseline to Week 13 in pruritus as measured by ItchRO(Pt) weekly average morning score. ItchRO scores range from 0 to 4; the higher score indicates increasing itch severity (0 = none; 4 = very severe).
Change From Baseline to Week 13/ET in ALT	Baseline (Day 0) to Week 13
Change From Baseline to Week 13/ET in Total Bilirubin	Baseline (Day 0) to Week 13

Trial Locations

Locations (10)

Children's Hospital Los Angeles; 🇺🇸 Los Angeles, California, United States

The Children's Memorial Health Institute; 🇵🇱 Warsaw, Poland

Hopital Femme Mere Enfant De Lyon; 🇫🇷 Bron, France

Children's Hospital Colorado; 🇺🇸 Aurora, Colorado, United States

Cincinnati Children's Hospital Medical Center; 🇺🇸 Cincinnati, Ohio, United States

The Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States

Birmingham Children's Hospital; 🇬🇧 Birmingham, West Midlands, United Kingdom

Children's Hospital of Pittsburgh of UPMC; 🇺🇸 Pittsburgh, Pennsylvania, United States

Leeds Teaching Hospital NHS Trust; 🇬🇧 Leeds, United Kingdom

Kings College Hospital; 🇬🇧 London, United Kingdom