New drug shows promise for babies with rare liver disorders

NCT ID NCT04729751

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

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Alagille syndrome bile duct disorder biliary tract disorder Cholestasis, progressive familial intrahepatic 1 liver disorder progressive familial intrahepatic cholestasis

As listed by the trial registrant

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

ALAGILLE SYNDROME CHOLESTATIC LIVER DISEASE PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS

Contacts and locations

Locations

Children Hospital LA

Los Angeles, California, 90027, United States
Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States
Cliniques Universitaires Saint-Luc

Brussels, Belgium
Consultorio de Joshue David Covarrubias Esquer

Zapopan, 45050, Mexico
Hopital Necker

Paris, France
Hôpital Kremlin Bicêtre

Le Kremlin-Bicêtre, France
Instytut Pomnik-Centrum Zdrowia Dziecka

Warsaw, Poland
King's College Hospital

London, United Kingdom
Medstar Georgetown University Hospital

Washington D.C., District of Columbia, 20007, United States
Ochsner Hospital for Children

New Orleans, Louisiana, 70121, United States
Seattle Children's Hospital

Seattle, Washington, 98105, United States
Sociedade Beneficente de Senhoras - Hospital Sírio-Libanês

São Paulo, 01308-000, Brazil
Texas Children's Hospital

Houston, Texas, 77030, United States
University of California - San Francisco

San Francisco, California, 94158, United States

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