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

Completed · Phase 2

Conditions studied: Cholestatic Liver Disease

In brief

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.

Key facts

Study ID

NCT04168385

Run by

Mirum Pharmaceuticals, Inc.

People needed

52

Starts

2020-01-16

Expected to finish

2024-09-04

Last updated by the study team

2025-12-08

Who can join

Age: 1 and older. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• A subject will be excluded from the study if any of the following exclusion criteria are met:
• Experienced an AE or SAE related to maralixibat during the lead-in protocol that led to permanent discontinuation of the subject from maralixibat.
• Any conditions or abnormalities (including laboratory abnormalities) which, in the opinion of the Investigator or Medical Monitor may compromise the safety of the subject or interfere with the subject participating in or completing the study.
• History of non-adherence to medical regimens, unreliability, medical condition, mental instability or cognitive impairment that, in the opinion of the Investigator or Sponsor medical monitor, could compromise the validity of informed consent, compromise the safety of the subject, or lead to non-adherence with the study protocol or inability to conduct the study procedures.

Where it is running

• Children'S Hospital Los Angeles — Los Angeles, California, United States
• Riley Hospital For Children — Indianapolis, Indiana, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Children Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
• Baylor College of Medicine/Texas Children's Hospital — Houston, Texas, United States
• Seattle Children's Hospital — Seattle, Washington, United States
• Children's Hospital Westmead — Westmead, New South Wales, Australia
• The Royal Children'S Hospital Melbourne — Parkville, Victoria, Australia
• Cliniques Universitaires Saint-Luc — Brussels, Belgium
• Hospital for Sick Children — Toronto, Canada
• Hopital Necker-Enfants Malades — Paris, France
• Hopital Kremlin Bicetre — Paris, France
• The Children's Memorial Health Institute — Warsaw, Poland
• Hospital Universitario La Paz- Hospital Materno Infantil — Madrid, Spain
• Birmingham Children's Hospital — Birmingham, United Kingdom
• Leeds Teaching Hospital NHS Trust — Leeds, United Kingdom
• Paediatric Liver Center, Kings College Hospital — London, United Kingdom

Full record on ClinicalTrials.gov

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