New drug shows promise for babies with rare liver disorders
NCT ID NCT04729751
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tested the safety of a drug called maralixibat in 27 infants under 12 months old with Alagille syndrome or progressive familial intrahepatic cholestasis (PFIC), two rare liver diseases that cause bile buildup. The drug is given as an oral solution and aims to reduce bile acids and improve liver function. The main goal was to see if it is safe and tolerable in this young age group.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
maralixibat (oral solution)
What this could lead to
If it works, this could offer a way to manage cholestatic liver disease in infants, reducing itching and bile acid buildup without immediate surgery.
What could go wrong
This is a small, early-phase study focused on safety, not proof of effectiveness. The drug may not work for all types of these rare diseases, and side effects are possible.
Disclaimer
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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.
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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Children Hospital LA
Los Angeles, California, 90027, United States
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Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
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Cliniques Universitaires Saint-Luc
Brussels, Belgium
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Consultorio de Joshue David Covarrubias Esquer
Zapopan, 45050, Mexico
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Hopital Necker
Paris, France
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Hôpital Kremlin Bicêtre
Le Kremlin-Bicêtre, France
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Instytut Pomnik-Centrum Zdrowia Dziecka
Warsaw, Poland
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King's College Hospital
London, United Kingdom
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Medstar Georgetown University Hospital
Washington D.C., District of Columbia, 20007, United States
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Ochsner Hospital for Children
New Orleans, Louisiana, 70121, United States
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Seattle Children's Hospital
Seattle, Washington, 98105, United States
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Sociedade Beneficente de Senhoras - Hospital Sírio-Libanês
São Paulo, 01308-000, Brazil
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Texas Children's Hospital
Houston, Texas, 77030, United States
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University of California - San Francisco
San Francisco, California, 94158, United States