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New drug livmarli tracked for Long-Term safety in kids with rare liver disorders

NCT ID NCT07290257

Recruiting now 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 follows 230 children with Alagille syndrome or progressive familial intrahepatic cholestasis (PFIC) who are taking Livmarli, a drug to reduce bile buildup and itching. Researchers will monitor side effects, liver function, and long-term outcomes like need for transplant. The goal is to see if Livmarli is safe and effective over years of use.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
Livmarli (maralixibat)
What this could lead to
If successful, this could confirm Livmarli as a safe long-term treatment to manage symptoms and slow liver damage in these rare childhood diseases.
What could go wrong
This is a low-intervention safety study, not a cure trial. It may show side effects or limited benefit, and results may not apply to all patients.

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 cholestasis Cholestasis, progressive familial intrahepatic 1 liver disorder progressive familial intrahepatic cholestasis Pruritus

As listed by the trial registrant

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

ALAGILLE SYNDROME PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS

Contacts and locations

Locations

  • 2nd Paediatric Clinic P &A Kyriakou Children's Hospital

    NOT_YET_RECRUITING

    Athens, 11527, Greece

  • AO Ospedale PAPA GIOVANNI XXIII

    RECRUITING

    Bergamo, Lombardy, 24126, Italy

  • Bicetre University Hospital

    RECRUITING

    Le Kremlin-Bicêtre, Île-de-France Region, 94270, France

  • CHU de Toulouse - Hôpital des Enfants

    RECRUITING

    Toulouse, Occitanie, 31049, France

  • Charite Berlin

    RECRUITING

    Berlin, 13353, Germany

  • Children's University Hospital Essen

    RECRUITING

    Essen, 45149, Germany

  • Cliniques Universitaires Saint Luc (UCLouvain)

    RECRUITING

    Brussels, 1200, Belgium

  • Hospices Civils de Lyon - Hopital Femme Mère Enfant

    RECRUITING

    Bron, Auvergne-Rhône-Alpes, 69500, France

  • Hospital Universitairo Vall D'Hebron

    RECRUITING

    Barcelona, Catalonia, 08035, Spain

  • Hospital Universitario La Paz

    RECRUITING

    Madrid, 28046, Spain

  • Istituto mediterraneo trapianti - ISMETT

    RECRUITING

    Palermo, Sicily, 90127, Italy

  • Unidade Local de Saude de Coimbra Paediatric Department Avenida Afonso Romăo

    NOT_YET_RECRUITING

    Coimbra, 3000-602, Portugal

  • University Hospital Gent (UZ Gent)

    RECRUITING

    Ghent, 9000, Belgium

  • University Hospital Hamburg-Eppendorf

    RECRUITING

    Hamburg, 20246, Germany

  • University Medical Center Groningen (UMCG)

    RECRUITING

    Groningen, 9713GZ, Netherlands

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