Liver drug maralixibat tested for Long-Term safety in rare disease patients
NCT ID NCT04168385
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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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Baylor College of Medicine/Texas Children's Hospital
Houston, Texas, 77030, United States
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Birmingham Children's Hospital
Birmingham, United Kingdom
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Children Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
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Children'S Hospital Los Angeles
Los Angeles, California, 90027, United States
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Children's Hospital Westmead
Westmead, New South Wales, 2145, Australia
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Cliniques Universitaires Saint-Luc
Brussels, Belgium
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Hopital Kremlin Bicetre
Paris, 94275, France
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Hopital Necker-Enfants Malades
Paris, 75015, France
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Hospital Universitario La Paz- Hospital Materno Infantil
Madrid, 261, Spain
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Hospital for Sick Children
Toronto, Canada
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Leeds Teaching Hospital NHS Trust
Leeds, LS1 3EX, United Kingdom
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Paediatric Liver Center, Kings College Hospital
London, SE5 9RS, United Kingdom
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Riley Hospital For Children
Indianapolis, Indiana, 46202, United States
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Seattle Children's Hospital
Seattle, Washington, 98105, United States
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The Children's Memorial Health Institute
Warsaw, 04-730, Poland
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The Royal Children'S Hospital Melbourne
Parkville, Victoria, 3052, Australia