Actively Recruiting
Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome in the European Union (LEAP-EU)
Led by Mirum Pharmaceuticals, Inc. · Updated on 2025-12-18
100
Participants Needed
13
Research Sites
273 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome in the European Union (LEAP-EU)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Provided informed consent and assent as applicable
- At least 2 months of age at the start of the study
- Clinically and/or genetically confirmed diagnosis of Alagille syndrome with pruritus due to chronic cholestasis
- Prescribed Livmarli at the time of study entry for the primary cohort
- Prescribed Livmarli prior to study entry for the supplemental cohort
You will not qualify if you...
- History of liver transplant
- Any contraindications to Livmarli as specified in its product information
- Any condition or abnormality that may interfere with participation or completion of the study, as judged by the investigator
- Received an investigational drug within 30 days before the first dose of Livmarli (previous participation in maralixibat studies or expanded-access programs is allowed)
- Lack of baseline data before starting Livmarli treatment (fewer than 2 values) for key safety tests (liver function, fat-soluble vitamins) and key efficacy measures (serum bile acids, pruritus)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 13 locations
1
Cliniques Universitaires Saint Luc (UCLouvain)
Brussels, Belgium, 1200
Not Yet Recruiting
2
University Hospital Gent (UZ Gent)
Ghent, Belgium, 9000
Not Yet Recruiting
3
Hospices Civils de Lyon - Hopital Femme Mère Enfant
Bron, Auvergne-Rhône-Alpes, France, 69500
Actively Recruiting
4
CHU de Toulouse - Hôpital des Enfants
Toulouse, Occitanie, France, 31049
Actively Recruiting
5
Bicetre University Hospital
Le Kremlin-Bicêtre, Île-de-France Region, France, 94270
Actively Recruiting
6
Charite Berlin
Berlin, Germany, 13353
Not Yet Recruiting
7
Children's University Hospital Essen
Essen, Germany, 45149
Not Yet Recruiting
8
University Hospital Hamburg-Eppendorf
Hamburg, Germany, 20246
Not Yet Recruiting
9
AO Ospedale PAPA GIOVANNI XXIII
Bergamo, Lombardy, Italy, 24126
Actively Recruiting
10
Istituto mediterraneo trapianti - ISMETT
Palermo, Sicily, Italy, 90127
Not Yet Recruiting
11
University Medical Center Groningen (UMCG)
Groningen, Netherlands, 9713GZ
Not Yet Recruiting
12
Hospital Universitairo Vall D'Hebron
Barcelona, Catalonia, Spain, 08035
Actively Recruiting
13
Hospital Universitario La Paz
Madrid, Spain, 28046
Actively Recruiting
Research Team
C
Clinical Trials Mirum
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
OTHER
Number of Arms
1
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