Actively Recruiting
Long-Term Low-Intervention Safety and Clinical Outcomes Study of Livmarli in Patients With Alagille Syndrome or Progressive Familial Intrahepatic Cholestasis in the European Union
Led by Mirum Pharmaceuticals, Inc. · Updated on 2026-06-03
230
Participants Needed
15
Research Sites
13 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the long-term safety, tolerability, and effectiveness of Livmarli treatment in patients diagnosed with either Alagille Syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC). The study focuses on monitoring possible liver toxicity and, for PFIC patients, the potential impact of prolonged exposure to propylene glycol. This open-label, low-intervention trial aims to collect clinical outcomes over an extended period to better understand the treatment's effects. Participants will receive Livmarli as part of their standard care throughout the study. The trial includes a primary cohort of at least 45 treatment-naïve ALGS patients and a supplemental ALGS cohort already using Livmarli. Additionally, a minimum of 130 PFIC patients treated with Livmarli will be enrolled. Treatment and assessments follow a defined schedule during the study period. Participants will undergo regular evaluations every 3 to 4 months for up to 5 years, including liver function tests, fat-soluble vitamin levels, international normalized ratio measurements, and monitoring of propylene glycol toxicity for PFIC patients. Adverse events are tracked up to 7 days after the last dose. Researchers will assess long-term clinical outcomes to understand treatment impact, with participants continuing their care according to standard practice during the study.
CONDITIONS
Brief Title
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)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Understand and provide informed consent and assent as applicable
- For Alagille Syndrome participants, be at least 2 months old at study start
- For Progressive Familial Intrahepatic Cholestasis participants, be at least 3 months old at study start
- Have a confirmed clinical or genetic diagnosis of Alagille Syndrome with pruritus due to chronic cholestasis or Progressive Familial Intrahepatic Cholestasis
- For Alagille Syndrome primary cohort, start Livmarli treatment at study entry
- For Alagille Syndrome supplemental cohort, be actively using Livmarli before study entry
- For Progressive Familial Intrahepatic Cholestasis participants, be prescribed Livmarli at or before study entry
You will not qualify if you...
- History of liver transplant
- Any contraindications to Livmarli as stated in the product information
- Any condition or abnormality that may interfere with participation or study completion as judged by the investigator
- Received an investigational drug within 30 days before the first Livmarli dose (except previous maralixibat studies or expanded-access programs)
- Received another IBAT inhibitor within 7 days before the first Livmarli dose
- Missing baseline data before starting Livmarli for key safety and efficacy parameters (less than 2 values)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 5 years
Participants are treated with Livmarli according to standard of care for the duration of the study.
Visits once every 3 to 4 months for assessments
Trial Site Locations
Total: 15 locations
1
Cliniques Universitaires Saint Luc (UCLouvain)
Brussels, Belgium, 1200
Actively Recruiting
2
University Hospital Gent (UZ Gent)
Ghent, Belgium, 9000
Actively 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
Actively Recruiting
7
Children's University Hospital Essen
Essen, Germany, 45149
Actively Recruiting
8
University Hospital Hamburg-Eppendorf
Hamburg, Germany, 20246
Actively Recruiting
9
2nd Paediatric Clinic P &A Kyriakou Children's Hospital
Athens, Greece, 11527
Not Yet Recruiting
10
AO Ospedale PAPA GIOVANNI XXIII
Bergamo, Lombardy, Italy, 24126
Actively Recruiting
11
Istituto mediterraneo trapianti - ISMETT
Palermo, Sicily, Italy, 90127
Actively Recruiting
12
University Medical Center Groningen (UMCG)
Groningen, Netherlands, 9713GZ
Actively Recruiting
13
Unidade Local de Saude de Coimbra Paediatric Department Avenida Afonso Romăo
Coimbra, Portugal, 3000-602
Not Yet Recruiting
14
Hospital Universitairo Vall D'Hebron
Barcelona, Catalonia, Spain, 08035
Actively Recruiting
15
Hospital Universitario La Paz
Madrid, Spain, 28046
Actively Recruiting
Research Team
C
Clinical Trials Mirum
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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