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)

NCT07290257 · tracked via the Priya Life Science Germany tracker

Sponsor

Mirum Pharmaceuticals, Inc

Phase

Phase 4

Started

2025-09-25

Last updated

2026-06-23

Condition(s) studied

Alagille SyndromeProgressive Familial Intrahepatic Cholestasis

Investigational drug(s) / intervention(s)

Livmarli Oral Product →

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

Study summary

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.

Eligibility

Sex

ALL

Min age

2 Months

Max age

—

Healthy volunteers

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

Primary outcome measure(s)

Adverse Events (AEs) — Up to 7 days after the last dose of Livmarli.
Number and proportion of participants with AEs.
Liver function tests (LFTs) - measuring enzyme activity or concentration — Once every 3-4 months, for 5 years.
Change in LFTs from Baseline: Total and direct bilirubin
Liver function tests (LFTs) - measuring enzyme activity or concentration — Once every 3-4 months, for 5 years.
Change in LFTs from Baseline: Aminotransferases and GGT.
Fat-Soluble Vitamins (FSV) Concentration — Once every 3-4 months, for 5 years.
Change in FSV levels (vitamins A, D, and E) from Baseline.
International Normalized Ratio (INR) — Once every 3-4 months, for 5 years.
Change in INR level from Baseline.
Long-Term Clinical Outcomes — 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.
Laboratory Test for Monitoring of Propylene Glycol Toxicity in Participants with PFIC — Once every 3-4 months, for 5 years.
Change in PG level from Baseline.

Trial sites (15)

Facility	City	Region	Status
Cliniques Universitaires Saint Luc (UCLouvain)	Brussels	Belgium	Recruiting
University Hospital Gent (UZ Gent)	Ghent	Belgium	Recruiting
Hospices Civils de Lyon - Hopital Femme Mère Enfant	Bron	Auvergne-Rhône-Alpes	Recruiting
CHU de Toulouse - Hôpital des Enfants	Toulouse	Occitanie	Recruiting
Bicetre University Hospital	Le Kremlin-Bicêtre	Île-de-France Region	Recruiting
Charite Berlin	Berlin	Germany	Recruiting
Children's University Hospital Essen	Essen	Germany	Recruiting
University Hospital Hamburg-Eppendorf	Hamburg	Germany	Recruiting
2nd Paediatric Clinic P &A Kyriakou Children's Hospital	Athens	Greece	Not Yet Recruiting
AO Ospedale PAPA GIOVANNI XXIII	Bergamo	Lombardy	Recruiting
Istituto mediterraneo trapianti - ISMETT	Palermo	Sicily	Recruiting
University Medical Center Groningen (UMCG)	Groningen	Netherlands	Recruiting
Unidade Local de Saude de Coimbra Paediatric Department Avenida Afonso Romăo	Coimbra	Portugal	Not Yet Recruiting
Hospital Universitairo Vall D'Hebron	Barcelona	Catalonia	Recruiting
Hospital Universitario La Paz	Madrid	Spain	Recruiting

More Mirum Pharmaceuticals, Inc. trials in Germany

Study to Evaluate Switching to Brelovitug for the Treatment of CHD in Participants Receiving Bulevirtide Recruiting

Evaluation of Maralixibat in Pruritus Associated With General Cholestatic Liver Disease (EXPAND) Active, not recruiting

A Trial Evaluating Brelovitug (BJT-778) vs Bulevirtide for the Treatment of Chronic Hepatitis Delta Infection (AZURE-2) Recruiting

A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC) Active, not recruiting

A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Biliary Cholangitis Active, not recruiting

Other trials for the same condition

Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome Active, not recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07290257 on ClinicalTrials.gov ↗ ← All trials in Germany