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

Phase 2

Completed

• Conditions: Cholestatic Liver Disease
• Interventions: Drug: Maralixibat

• Registration Number: NCT04168385
• Lead Sponsor: Mirum Pharmaceuticals, Inc.

Brief 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 will receive maralixibat in this study.

Study Timeline

• Study First Submitted: 2019-10-01
• Study First Submitted QC: 2019-11-17
• Study First Posted: 2019-11-19
• Study Start: 2020-01-16
• Primary Completion: 2024-09-04
• Study Completion: 2024-09-04
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-03
• Last Update Posted: 2025-03-06

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 52

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Maralixibat	Maralixibat	Participants will all receive Maralixibat oral solution

Primary Outcome Measures

Name	Time	Method
Frequency of reported adverse events AEs	From baseline through study completion, up to approximately 3 years	Treatment-emergent AEs (TEAEs) will be defined as AEs that start or deteriorate on or after the first dose of study medication and no later than 14 days following the last dose of study medication or reported through the End of Trial ( EOT/ET) Visit. In this study, all AEs will be considered TEAEs.

Secondary Outcome Measures

Name	Time	Method
Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ frequency score (ALGS and PFIC)	From baseline through study completion, up to approximately 3 years
Evaluate the long-term effect of maralixibat on pruritus severity using in the Clinician Scratch Scale (CSS) as change from baseline over the course of the study	From baseline through study completion, up to approximately 3 years
Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ severity score (ALGS and PFIC)	From baseline through study completion, up to approximately 3 years
Evaluate the long-term effect of maralixibat on serum bile acid levels	From baseline through study completion, up to approximately 3 years
Evaluate the long-term effect of maralixibat on time to liver-associated outcomes reported as a mean change from baseline (i.e., partial external biliary diversion [PEBD] or liver transplantation)	From baseline to occurrence of liver associated event, up to approximately 3 years
Change from maralixibat baseline over the course of the study in mean total serum bilirubin	Change from baseline through study completion, up to approximately 3 years
Evaluate the long-term effects of maralixibat on growth	Through study completion, up to approximately 3 years	Change from baseline in height and weight, summarized as a Z-score over the course of the study

Trial Locations

Locations (17)

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

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

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

Hopital Necker-Enfants Malades; 🇫🇷 Paris, France

Children Hospital of Pittsburgh; 🇺🇸 Pittsburgh, Pennsylvania, United States

Baylor College of Medicine/Texas Children's Hospital; 🇺🇸 Houston, Texas, United States

Riley Hospital For Children; 🇺🇸 Indianapolis, Indiana, United States

Hospital Universitario La Paz- Hospital Materno Infantil; 🇪🇸 Madrid, Spain

Paediatric Liver Center, Kings College Hospital; 🇬🇧 London, United Kingdom

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

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

Children's Hospital Westmead; 🇦🇺 Westmead, New South Wales, Australia

Seattle Children's Hospital; 🇺🇸 Seattle, Washington, United States

The Royal Children'S Hospital Melbourne; 🇦🇺 Parkville, Victoria, Australia

Cliniques Universitaires Saint-Luc; 🇧🇪 Brussels, Belgium

Hospital for Sick Children; 🇨🇦 Toronto, Canada

Hopital Kremlin Bicetre; 🇫🇷 Paris, France