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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.
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
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