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)
Recruiting now · Phase 4
Conditions studied: Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis
In brief
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 facts
- Study ID
- NCT07290257
- Run by
- Mirum Pharmaceuticals, Inc.
- People needed
- 230
- Starts
- 2025-09-25
- Expected to finish
- 2030-12-22
- Last updated by the study team
- 2026-06-23
Who can join
Age: 0 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Understand and execute an Informed consent and assent (as applicable)
- For participants with ALGS ≥2 months of age at Day 1
- For participants with PFIC, ≥3 months of age at Day 1
- A clinically and/or genetically confirmed ALGS diagnosis with pruritus secondary to chronic cholestasis, or a clinically and/or genetically confirmed PFIC diagnosis
- For the ALGS primary cohort: Initiation of Livmarli at the time of study entry
- For the ALGS supplemental cohort, : Actively using Livmarli prior to study entry
- For participants with PFIC: Prescribed Livmarli at the time of study entry or prior to study entry
You may not qualify if…
- History of Liver Transplant
- Any Livmarli contraindications (as per SmPC)
- Any condition or abnormality that, in the opinion of the investigator, may interfere with the participation in or completion of the study
- Received an investigational drug within 30 days before the first dose of Livmarli (Participation in previous maralixibat studies or expanded-access programs is acceptable.)
- Received another IBAT inhibitor within 7 days before the first dose of Livmarli
- Baseline data before start of treatment of Livmarli are unavailable (<2 values before treatment) for key safety (LFTs, FSV laboratory results) and key efficacy (sBA, pruritus) parameter
Where it is running
- Hospital Universitario La Paz — Madrid, Spain (enrolling)
- University Hospital Gent (UZ Gent) — Ghent, Belgium (enrolling)
- Hospices Civils de Lyon - Hopital Femme Mère Enfant — Bron, Auvergne-Rhône-Alpes, France (enrolling)
- CHU de Toulouse - Hôpital des Enfants — Toulouse, Occitanie, France (enrolling)
- Bicetre University Hospital — Le Kremlin-Bicêtre, Île-de-France Region, France (enrolling)
- Charite Berlin — Berlin, Germany (enrolling)
- Children's University Hospital Essen — Essen, Germany (enrolling)
- University Hospital Hamburg-Eppendorf — Hamburg, Germany (enrolling)
- Hospital Universitairo Vall D'Hebron — Barcelona, Catalonia, Spain (enrolling)
- Cliniques Universitaires Saint Luc (UCLouvain) — Brussels, Belgium (enrolling)
- AO Ospedale PAPA GIOVANNI XXIII — Bergamo, Lombardy, Italy (enrolling)
- Istituto mediterraneo trapianti - ISMETT — Palermo, Sicily, Italy (enrolling)
- University Medical Center Groningen (UMCG) — Groningen, Netherlands (enrolling)
- 2nd Paediatric Clinic P &A Kyriakou Children's Hospital — Athens, Greece
- Unidade Local de Saude de Coimbra Paediatric Department Avenida Afonso Romăo — Coimbra, Portugal
Full record on ClinicalTrials.gov
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