Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome in the European Union (LEAP-EU)
Status: Recruiting
Location: See all (13) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 4
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 2 months
Healthy Volunteers: f
View:
• Informed consent and assent (as applicable)
• ≥2 months of age at Day 1
• A clinically and/or genetically confirmed ALGS diagnosis with pruritus secondary to chronic cholestasis
• For the primary cohort, prescribed Livmarli at time of study entry
• For the supplemental cohort, prescribed Livmarli prior to study entry
Locations
Other Locations
Belgium
Cliniques Universitaires Saint Luc (UCLouvain)
NOT_YET_RECRUITING
Brussels
University Hospital Gent (UZ Gent)
NOT_YET_RECRUITING
Ghent
France
Hospices Civils de Lyon - Hopital Femme Mère Enfant
RECRUITING
Bron
Bicetre University Hospital
RECRUITING
Le Kremlin-bicêtre
CHU de Toulouse - Hôpital des Enfants
RECRUITING
Toulouse
Germany
Charite Berlin
NOT_YET_RECRUITING
Berlin
Children's University Hospital Essen
NOT_YET_RECRUITING
Essen
University Hospital Hamburg-Eppendorf
NOT_YET_RECRUITING
Hamburg
Italy
AO Ospedale PAPA GIOVANNI XXIII
RECRUITING
Bergamo
Istituto mediterraneo trapianti - ISMETT
NOT_YET_RECRUITING
Palermo
Netherlands
University Medical Center Groningen (UMCG)
NOT_YET_RECRUITING
Groningen
Spain
Hospital Universitairo Vall D'Hebron
RECRUITING
Barcelona
Hospital Universitario La Paz
RECRUITING
Madrid
Contact Information
Primary
Clinical Trials Mirum
clinicaltrials@mirumpharma.com
+16506674085
Time Frame
Start Date: 2025-09-25
Estimated Completion Date: 2030-12-22
Participants
Target number of participants: 100
Treatments
Experimental: Patients with Alagile Syndrome treated with Livmarli
Primary cohort with a minimum of 45 previously untreated (treatment naïve). Supplemental cohort of participants who have previously received Livmarli.
Related Therapeutic Areas
Sponsors
Leads: Mirum Pharmaceuticals, Inc.