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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE4

Total Enrollment

100 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-09-25

Study Completion Date

2030-12-22

Brief Summary

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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.

Detailed Description

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This is a multicenter, open-label, low-intervention clinical study in participants diagnosed with Alagille syndrome (ALGS) who are treated with Livmarli for cholestatic pruritus.

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

Conditions

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Alagille Syndrome

Keywords

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

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

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.

Primary Study Purpose

OTHER

Blinding Strategy

NONE

Study Groups

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Patients with Alagile Syndrome treated with Livmarli

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

Group Type EXPERIMENTAL

Livmarli Oral Product

Intervention Type DRUG

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

Interventions

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Livmarli Oral Product

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

Intervention Type DRUG

Other Intervention Names

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Maralixibat

Eligibility Criteria

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Inclusion Criteria

* 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

Exclusion Criteria

* 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

Minimum Eligible Age

2 Months

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

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Clinical Trials Mirum

Role: CONTACT

Phone: +16506674085

Identifier Type: -

Identifier Source: org_study_id

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

Study Results

Basic Information

Brief Summary

Detailed Description

Conditions

Keywords

Study Design

Study Groups

Patients with Alagile Syndrome treated with Livmarli

Livmarli Oral Product

Interventions

Livmarli Oral Product

Other Intervention Names

Eligibility Criteria

Inclusion Criteria

Exclusion Criteria

Sponsors

Mirum Pharmaceuticals, Inc.

Responsible Party

Locations

Cliniques Universitaires Saint Luc (UCLouvain)

University Hospital Gent (UZ Gent)

Hospices Civils de Lyon - Hopital Femme Mère Enfant

CHU de Toulouse - Hôpital des Enfants

Bicetre University Hospital

Charite Berlin

Children's University Hospital Essen

University Hospital Hamburg-Eppendorf

AO Ospedale PAPA GIOVANNI XXIII

Istituto mediterraneo trapianti - ISMETT

University Medical Center Groningen (UMCG)

Hospital Universitairo Vall D'Hebron

Hospital Universitario La Paz

Countries

Central Contacts

Clinical Trials Mirum

Facility Contacts

Stephenne Xavier

Ruth De Bruyne

Mathias Ruiz

Nolwenn Laborde

Emmanuel Gonzales

Philip Bufler

Elke Lainka

Sebastian Schulz-Jürgensen

Lorenzo D'Antiga

Giusy Ranucci

Henkjan Verkade

Jesus Quintero

Esteban Frauca Remacha

Other Identifiers

MRX-803