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Liver drug maralixibat tested for Long-Term safety in rare disease patients

NCT ID NCT04168385

Completed Disease control Sponsor: Mirum Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study looked at the long-term safety of a drug called maralixibat in 52 people with cholestatic liver disease, including Alagille Syndrome and other rare conditions. Participants had already taken maralixibat in an earlier study. Researchers tracked side effects and how well the drug controlled itching over time.

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Conditions

The condition(s) this trial relates to.

Alagille syndrome bile duct disorder biliary atresia biliary tract disorder Digestive System Diseases liver disorder progressive familial intrahepatic cholestasis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

CHOLESTATIC LIVER DISEASE

Contacts and locations

Locations

  • Baylor College of Medicine/Texas Children's Hospital

    Houston, Texas, 77030, United States

  • Birmingham Children's Hospital

    Birmingham, United Kingdom

  • Children Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States

  • Children'S Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital Westmead

    Westmead, New South Wales, 2145, Australia

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Cliniques Universitaires Saint-Luc

    Brussels, Belgium

  • Hopital Kremlin Bicetre

    Paris, 94275, France

  • Hopital Necker-Enfants Malades

    Paris, 75015, France

  • Hospital Universitario La Paz- Hospital Materno Infantil

    Madrid, 261, Spain

  • Hospital for Sick Children

    Toronto, Canada

  • Leeds Teaching Hospital NHS Trust

    Leeds, LS1 3EX, United Kingdom

  • Paediatric Liver Center, Kings College Hospital

    London, SE5 9RS, United Kingdom

  • Riley Hospital For Children

    Indianapolis, Indiana, 46202, United States

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • The Children's Memorial Health Institute

    Warsaw, 04-730, Poland

  • The Royal Children'S Hospital Melbourne

    Parkville, Victoria, 3052, Australia

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