RecruitingPhase 4NCT07290257

Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome or Progressive Familial Intrahepatic Cholestasis in the European Union (LEAP-EU)

Sponsor

Mirum Pharmaceuticals, Inc.

Enrollment

230 participants

Start Date

Sep 25, 2025

Study Type

INTERVENTIONAL

Conditions

Progressive Familial Intrahepatic Cholestasis Alagille Syndrome

Summary

In patients with Alagille syndrome (ALGS), the key objectives are to evaluate the tolerability, long-term safety (including possible liver toxicity) and long-term efficacy of Livmarli treatment. In patients with Progressive Familial Intrahepatic Cholestasis (PFIC), the key objectives are to evaluate tolerability, long-term safety (including possible liver toxicity and potential impact of chronic exposure to propylene glycol (PG)), long-term efficacy, and growth and development.

Eligibility

Min Age: 2 Months

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Livmarli Oral Product for people with alagille syndrome and progressive familial intrahepatic cholestasis. The study is currently recruiting participants at 15 locations.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGLivmarli Oral Product

Participants will be treated according to standard of care for the duration of the study and Schedule of Assessments.

Locations(15)

Cliniques Universitaires Saint Luc (UCLouvain)

Brussels, Belgium

University Hospital Gent (UZ Gent)

Ghent, Belgium

Hospices Civils de Lyon - Hopital Femme Mère Enfant

Bron, Auvergne-Rhône-Alpes, France

CHU de Toulouse - Hôpital des Enfants

Toulouse, Occitanie, France

Bicetre University Hospital

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

Charite Berlin

Berlin, Germany

Children's University Hospital Essen

Essen, Germany

University Hospital Hamburg-Eppendorf

Hamburg, Germany

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

Athens, Greece

AO Ospedale PAPA GIOVANNI XXIII

Bergamo, Lombardy, Italy

Istituto mediterraneo trapianti - ISMETT

Palermo, Sicily, Italy

University Medical Center Groningen (UMCG)

Groningen, Netherlands

Unidade Local de Saude de Coimbra Paediatric Department Avenida Afonso Romăo

Coimbra, Portugal

Hospital Universitairo Vall D'Hebron

Barcelona, Catalonia, Spain

Hospital Universitario La Paz

Madrid, Spain

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

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NCT07290257

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