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RECRUITING
NCT07290257
PHASE4

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.

View on ClinicalTrials.gov

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.

Key Details

Gender

All

Age Range

2 Months - Any

Study Type

INTERVENTIONAL

Enrollment

230

Start Date

2025-09-25

Completion Date

2030-12-22

Last Updated

2026-06-23

Healthy Volunteers

No

Interventions

DRUG

Livmarli 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