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NCT Number: NCT07290257

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)

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.

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Key information

Conditions

Alagille Syndrome Abnormalities, Multiple Bile Duct Diseases Biliary Tract Diseases Cardiovascular Abnormalities Cardiovascular Diseases Cholestasis Cholestasis, Intrahepatic Cholestasis, progressive familial intrahepatic 1 Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Heart Defects, Congenital Liver Diseases Pathological Conditions, Signs and Symptoms Progressive Familial Intrahepatic Cholestasis Pruritus Signs and Symptoms Skin Diseases Skin Manifestations Skin and Connective Tissue Diseases

Age range

2 month and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Cliniques Universitaires Saint Luc (UCLouvain), Brussels, Belgium

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About this study

This is a multicenter, open-label, low-intervention clinical study in participants diagnosed with either ALGS with cholestatic pruritus or PFIC who are treated with Livmarli.

Participants will be treated according to standard of care for the duration of the study.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Livmarli Oral Product

Drug

Participants will be treated according to standard of care for the duration of the study and Schedule of Assessments.

Other names: Maralixibat

Primary outcomes

  1. Adverse Events (AEs)

    Time frame: Up to 7 days after the last dose of Livmarli.

    Number and proportion of participants with AEs.

  2. Liver function tests (LFTs) - measuring enzyme activity or concentration

    Time frame: Once every 3-4 months, for 5 years.

    Change in LFTs from Baseline: Total and direct bilirubin

  3. Liver function tests (LFTs) - measuring enzyme activity or concentration

    Time frame: Once every 3-4 months, for 5 years.

    Change in LFTs from Baseline: Aminotransferases and GGT.

  4. Fat-Soluble Vitamins (FSV) Concentration

    Time frame: Once every 3-4 months, for 5 years.

    Change in FSV levels (vitamins A, D, and E) from Baseline.

  5. International Normalized Ratio (INR)

    Time frame: Once every 3-4 months, for 5 years.

    Change in INR level from Baseline.

  6. Long-Term Clinical Outcomes

    Time frame: Once every 3-4 months, for 5 years.

    Number of participants with surgical biliary diversion, liver transplantation, liver transplant waitlist status change, clinically evident portal hypertension, complications of liver cirrhosis, liver carcinoma, liver decompensation, and death.

  7. Laboratory Test for Monitoring of Propylene Glycol Toxicity in Participants with PFIC

    Time frame: Once every 3-4 months, for 5 years.

    Change in PG level from Baseline.

Study contacts

Contact information is provided by the study sponsor or research team.

Clinical Trials Mirum

CONTACT

[email protected]

+16506674085

Sponsors and collaborators

Lead sponsor

Mirum Pharmaceuticals, Inc.

Industry

Registry information

Important dates

Study start
2025
Primary completion
2030
Study completion
2030
First posted
Dec 18, 2025
Registry last updated
Jun 23, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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