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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)

RecruitingPhase 4Ages 2 Months - anyAllMirum Pharmaceuticals, Inc.
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In plain words

What the trial is, and whether it could suit you

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.

Sign in to get the eligibility rules as simple questions to take to your doctor.

📋 Eligibility rules (as published)
Inclusion Criteria: * 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 Exclusion Criteria: * 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
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What it tests

  • Livmarli Oral Product
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Where

15 sites
Cliniques Universitaires Saint Luc (UCLouvain)
Brussels, Belgium
University Hospital Gent (UZ Gent)
Ghent, Belgium
Hospices Civils de Lyon - Hopital Femme Mère Enfant
Bron, France
CHU de Toulouse - Hôpital des Enfants
Toulouse, France
Bicetre University Hospital
Le Kremlin-Bicêtre, France
Charite Berlin
Berlin, Germany
Children's University Hospital Essen
Essen, Germany
University Hospital Hamburg-Eppendorf
Hamburg, Germany

…and 7 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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