Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome in the European Union (LEAP-EU)

NCT07290257 | Recruiting Phase 4

• 1 other identifier: MRX-803
• Study Type: interventional
• Target: 100
• Locations: 6 countries
• Sites: 13

Brief Summary

The key objectives of this low-intervention clinical study are to evaluate tolerability, long-term safety and long-term efficacy for patients with Alagille syndrome (ALGS) who are prescribed Livmarli.

Conditions

Alagille Syndrome

Keywords

ALGS; Liver Diseases; Alagille Syndrome; Livmarli; Maralixibat; Cholestasis; Pruritus; Itching; IBAT inhibitor

Outcome Measures

Primary Outcomes (5)

• Adverse Events (AEs)

  Number and proportion of participants with AEs.

  Up to 7 days after the last dose of Livmarli.

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

  Change in LFTs from Baseline: Total and direct bilirubin, aminotransferases, GGT, and alkaline phosphatase.

  Once every 3-4 months, for 5 years.

• Fat-Soluble Vitamins (FSV) Concentration

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

  Once every 3-4 months, for 5 years.

• International Normalized Ratio (INR)

  Change in INR level from Baseline.

  Once every 3-4 months, for 5 years.

• Long-Term Clinical Outcomes

  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.

Study Arms (1)

Patients with Alagile Syndrome treated with Livmarli

EXPERIMENTAL

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

Drug: Livmarli Oral Product

Interventions

Livmarli Oral Product DRUG

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

Also known as: Maralixibat

Patients with Alagile Syndrome treated with Livmarli

Eligibility Criteria

• Age: 2 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Informed consent and assent (as applicable)
• ≥2 months of age at Day 1
• A clinically and/or genetically confirmed ALGS diagnosis with pruritus secondary to chronic cholestasis
• For the primary cohort, prescribed Livmarli at time of study entry
• For the supplemental cohort, prescribed Livmarli prior to study entry

You may not qualify if:

• History of Liver Transplant
• Any contraindications against Livmarli (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.)
• 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

Sponsors & Collaborators

• Mirum Pharmaceuticals, Inc.: lead

Study Sites (13)

Cliniques Universitaires Saint Luc (UCLouvain)

Brussels, 1200, Belgium

NOT YET RECRUITING

University Hospital Gent (UZ Gent)

Ghent, 9000, Belgium

NOT YET RECRUITING

Hospices Civils de Lyon - Hopital Femme Mère Enfant

Bron, Auvergne-Rhône-Alpes, 69500, France

RECRUITING

CHU de Toulouse - Hôpital des Enfants

Toulouse, Occitanie, 31049, France

RECRUITING

Bicetre University Hospital

Le Kremlin-Bicêtre, Île-de-France Region, 94270, France

RECRUITING

Charite Berlin

Berlin, 13353, Germany

NOT YET RECRUITING

Children's University Hospital Essen

Essen, 45149, Germany

NOT YET RECRUITING

University Hospital Hamburg-Eppendorf

Hamburg, 20246, Germany

NOT YET RECRUITING

AO Ospedale PAPA GIOVANNI XXIII

Bergamo, Lombardy, 24126, Italy

RECRUITING

Istituto mediterraneo trapianti - ISMETT

Palermo, Sicily, 90127, Italy

NOT YET RECRUITING

University Medical Center Groningen (UMCG)

Groningen, 9713GZ, Netherlands

NOT YET RECRUITING

Hospital Universitairo Vall D'Hebron

Barcelona, Catalonia, 08035, Spain

RECRUITING

Hospital Universitario La Paz

Madrid, 28046, Spain

RECRUITING

MeSH Terms

Conditions

Alagille Syndrome; Liver Diseases; Cholestasis; Pruritus

Interventions

maralixibat

Condition Hierarchy (Ancestors)

Cholestasis, Intrahepatic; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Skin Diseases; Skin and Connective Tissue Diseases; Skin Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Central Study Contacts

Clinical Trials Mirum

CONTACT

+16506674085; clinicaltrials@mirumpharma.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: The study will enroll a primary cohort with a minimum of 45 previously untreated (treatment naïve) patients with ALGS including at least 5 participants \<1 year of age, 30 participants between 1 and 5 years of age, and 10 participants \>5 years of age. A supplemental cohort of participants who have previously received Livmarli will also be enrolled. There is no enrollment limit for supplementary cohort. For admin purposes, the number has been set to 55 participants.

Study Record Dates

• First Submitted: September 17, 2025
• First Posted: December 18, 2025
• Study Start: September 25, 2025
• Primary Completion (Estimated): September 22, 2030
• Study Completion (Estimated): December 22, 2030
• Last Updated: December 18, 2025

Record last verified: 2025-12

Locations

NL (1); BE (2); FR (3); ES (2); DE (3); IT (2)