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)

June 1, 2026 updated by: Mirum Pharmaceuticals, Inc.

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

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.

Study Type

Interventional

Enrollment (Estimated)

230

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Belgium
      • Brussels, Belgium, 1200
        • Recruiting
        • Cliniques Universitaires Saint Luc (UCLouvain)
        • Contact:
      • Ghent, Belgium, 9000
        • Recruiting
        • University Hospital Gent (UZ Gent)
        • Contact:
  • France
    • Auvergne-Rhône-Alpes
      • Bron, Auvergne-Rhône-Alpes, France, 69500
        • Recruiting
        • Hospices Civils de Lyon - Hopital Femme Mère Enfant
        • Contact:
    • Occitanie
      • Toulouse, Occitanie, France, 31049
        • Recruiting
        • CHU de Toulouse - Hôpital des Enfants
        • Contact:
    • Île-de-France Region
      • Le Kremlin-Bicêtre, Île-de-France Region, France, 94270
        • Recruiting
        • Bicetre University Hospital
        • Contact:
  • Germany
      • Berlin, Germany, 13353
      • Essen, Germany, 45149
        • Recruiting
        • Children's University Hospital Essen
        • Contact:
      • Hamburg, Germany, 20246
        • Recruiting
        • University Hospital Hamburg-Eppendorf
        • Contact:
  • Greece
      • Athens, Greece, 11527
        • Not yet recruiting
        • 2nd Paediatric Clinic P &A Kyriakou Children's Hospital
        • Contact:
          • Aglaia Zellos
  • Italy
    • Lombardy
      • Bergamo, Lombardy, Italy, 24126
    • Sicily
      • Palermo, Sicily, Italy, 90127
        • Recruiting
        • Istituto mediterraneo trapianti - ISMETT
        • Contact:
  • Netherlands
      • Groningen, Netherlands, 9713GZ
        • Recruiting
        • University Medical Center Groningen (UMCG)
        • Contact:
  • Portugal
      • Coimbra, Portugal, 3000-602
        • Not yet recruiting
        • Unidade Local de Saude de Coimbra Paediatric Department Avenida Afonso Romăo
        • Contact:
          • Isabel Goncalves Costa
  • Spain

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Other
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Livmarli

ALGS: Primary cohort with a minimum of 45 previously untreated (treatment naïve). Supplemental cohort of participants who have previously received Livmarli.

PFIC: Patients with Progressive Familial Intrahepatic Cholestasis treated with Livmarli. The study will enroll a minimum of 130 patients with PFIC
Drug: Livmarli Oral Product
Participants will be treated according to standard of care for the duration of the study and Schedule of Assessments.
Other Names:
  • Maralixibat

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Adverse Events (AEs)
Time Frame: Up to 7 days after the last dose of Livmarli.
Number and proportion of participants with AEs.
Up to 7 days after the last dose of Livmarli.
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.
Once every 3-4 months, for 5 years.
International Normalized Ratio (INR)
Time Frame: Once every 3-4 months, for 5 years.
Change in INR level from Baseline.
Once every 3-4 months, for 5 years.
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.
Once every 3-4 months, for 5 years.
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, aminotransferases and GGT.
Once every 3-4 months, for 5 years.
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.
Once every 3-4 months, for 5 years.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Mirum Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 25, 2025

Primary Completion (Estimated)

September 22, 2030

Study Completion (Estimated)

December 22, 2030

Study Registration Dates

First Submitted

September 17, 2025

First Submitted That Met QC Criteria

December 4, 2025

First Posted (Actual)

December 18, 2025

Study Record Updates

Last Update Posted (Actual)

June 3, 2026

Last Update Submitted That Met QC Criteria

June 1, 2026

Last Verified

June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MRX-803

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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