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Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome in the European Union (LEAP-EU)
Sponsor: Mirum Pharmaceuticals, Inc.
Summary
The key objectives of this low-intervention clinical study are to evaluate tolerability, long-term safety and long-term efficacy for patients with Alagille syndrome (ALGS) who are prescribed Livmarli.
Key Details
Gender
All
Age Range
2 Months - Any
Study Type
INTERVENTIONAL
Enrollment
100
Start Date
2025-09-25
Completion Date
2030-12-22
Last Updated
2025-12-18
Healthy Volunteers
No
Conditions
Interventions
Livmarli Oral Product
Participants will be treated according to standard of care for the duration of the study and Schedule of Assessments.
Locations (13)
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
AO Ospedale PAPA GIOVANNI XXIII
Bergamo, Lombardy, Italy
Istituto mediterraneo trapianti - ISMETT
Palermo, Sicily, Italy
University Medical Center Groningen (UMCG)
Groningen, Netherlands
Hospital Universitairo Vall D'Hebron
Barcelona, Catalonia, Spain
Hospital Universitario La Paz
Madrid, Spain