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Study identifier: NCT07290257 Synced from ClinicalTrials.gov · July 29, 2026
● Recruiting

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)

Condition: Alagille Syndrome · Progressive Familial Intrahepatic Cholestasis  ·  Sponsor: Mirum Pharmaceuticals, Inc.

PhasePhase 4
Planned participants230
Who can joinAll sexes, 2 Months to no upper limit
Healthy volunteersNo

About this study

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.

This description comes directly from the study's public registry record.

Talk to the study team

Clinical Trials Mirum  ·  +16506674085  ·  clinicaltrials@mirumpharma.com

Always discuss trial participation with your own doctor first.

Locations (15)

Cliniques Universitaires Saint Luc (UCLouvain)Brussels, BelgiumRecruiting
University Hospital Gent (UZ Gent)Ghent, BelgiumRecruiting
Hospices Civils de Lyon - Hopital Femme Mère EnfantBron, Auvergne-Rhône-Alpes, FranceRecruiting
CHU de Toulouse - Hôpital des EnfantsToulouse, Occitanie, FranceRecruiting
Bicetre University HospitalLe Kremlin-Bicêtre, Île-de-France Region, FranceRecruiting
Charite BerlinBerlin, GermanyRecruiting
Children's University Hospital EssenEssen, GermanyRecruiting
University Hospital Hamburg-EppendorfHamburg, GermanyRecruiting
2nd Paediatric Clinic P &A Kyriakou Children's HospitalAthens, GreeceNot Yet Recruiting
AO Ospedale PAPA GIOVANNI XXIIIBergamo, Lombardy, ItalyRecruiting
Istituto mediterraneo trapianti - ISMETTPalermo, Sicily, ItalyRecruiting
University Medical Center Groningen (UMCG)Groningen, NetherlandsRecruiting

+ 3 more locations — full list on the registry record.

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Source record: clinicaltrials.gov/study/NCT07290257