Hope for rare liver disease: drug aims to ease severe itching in children
NCT ID NCT04185363
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests the long-term safety of maralixibat, an oral medication, in children with progressive familial intrahepatic cholestasis (PFIC), a rare liver disease that causes intense itching. Participants who completed a prior study will receive maralixibat twice daily. Researchers will monitor side effects and track changes in itching severity over time.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
maralixibat
What this could lead to
If successful, this could confirm maralixibat as a safe long-term treatment to control itching and improve quality of life in children with PFIC.
What could go wrong
This is an extension study, so results depend on earlier findings. The drug may not work for everyone, and long-term side effects are still being evaluated.
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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Advent Health
Orlando, Florida, 32803, United States
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Azienda Ospedaliera Papa Giovanni XXIII - Unita di Pediatria
Bergamo, 24127, Italy
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Birmingham Children's Hospital
Birmingham, United Kingdom
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CHU de Toulouse - Hôpital des Enfants
Toulouse, 31059, France
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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 at Montefiore
The Bronx, New York, 10461, United States
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Cincinnati Children's Hospital
Cincinnati, Ohio, 45229, United States
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Cliniques Universitaires Saint-Luc
Brussels, Belgium
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Consultario de Joshue David Covarrubias Esquer
Zapopan, Mexico
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Fundacion Cardioinfantil
Bogotá, Colombia
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Groupement Hospitalier Est Hopital, Femme Mère Enfant de Lyon
Lyon, France
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Hospital Italiano de Buenos Aires
Buenos Aires, Argentina
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Hotel Dieu de France, Alfred Naccache
Beirut, Lebanon
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Instytut Pomnik Centrum, Zdrowia Dziecka
Warsaw, Poland
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KK Women's and Children's Hospital
Singapore, Singapore
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King's College Hospital NHS Foundation Trust
London, United Kingdom
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Koc University Hospital
Istanbul, 34010, Turkey (Türkiye)
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Medical University of South Carolina
Charleston, South Carolina, 29425, United States
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Medizinische Hochschule
Hanover, Germany
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Medizinische Universität Wien, Universitätsklinik für Kinder- und Jugendheilkunde
Vienna, Austria
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Medstar Georgetown University Hospital
Washington D.C., District of Columbia, 20007, United States
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Ospedale Pediatrico bambino Gesu'
Roma, Italy
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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, Brazil
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University of Alberta - Women and Children's Health Research Institute
Edmonton, Alberta, Canada
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University of Texas Southwestern Medical Center
Dallas, Texas, 75390, United States
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University of Texas, Health Science Center San Antonio
San Antonio, Texas, 78229, United States