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New drug livmarli tracked for Long-Term safety in kids with rare liver disorders

NCT ID NCT07290257

Disease control Sponsor: Mirum Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study follows 230 children with Alagille syndrome or progressive familial intrahepatic cholestasis (PFIC) who are taking Livmarli, a drug to reduce bile buildup and itching. Researchers will monitor side effects, liver function, and long-term outcomes like need for transplant. The goal is to see if Livmarli is safe and effective over years of use.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Livmarli (maralixibat)
What this could lead to
If successful, this could confirm Livmarli as a safe long-term treatment to manage symptoms and slow liver damage in these rare childhood diseases.
What could go wrong
This is a low-intervention safety study, not a cure trial. It may show side effects or limited benefit, and results may not apply to all patients.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 4

Runs after approval, following long-term safety and how well the treatment works in everyday use.

Participants

About 230 people

The number the study aims to enrol. It can still change while the study runs.

Started

Sep 2025

Expected to finish

Dec 2030

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 months and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

Alagille syndrome cholestasis Cholestasis, progressive familial intrahepatic 1 liver disorder progressive familial intrahepatic cholestasis Pruritus

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Alagille syndrome Progressive familial intrahepatic cholestasis

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    15 sites in 8 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • 2nd Paediatric Clinic P &A Kyriakou Children's Hospital

    NOT_YET_RECRUITING

    Athens, 11527, Greece

  • AO Ospedale PAPA GIOVANNI XXIII

    RECRUITING

    Bergamo, Lombardy, 24126, Italy

  • Bicetre University Hospital

    RECRUITING

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

  • CHU de Toulouse - Hôpital des Enfants

    RECRUITING

    Toulouse, Occitanie, 31049, France

  • Charite Berlin

    RECRUITING

    Berlin, 13353, Germany

  • Children's University Hospital Essen

    RECRUITING

    Essen, 45149, Germany

  • Cliniques Universitaires Saint Luc (UCLouvain)

    RECRUITING

    Brussels, 1200, Belgium

  • Hospices Civils de Lyon - Hopital Femme Mère Enfant

    RECRUITING

    Bron, Auvergne-Rhône-Alpes, 69500, France

  • Hospital Universitairo Vall D'Hebron

    RECRUITING

    Barcelona, Catalonia, 08035, Spain

  • Hospital Universitario La Paz

    RECRUITING

    Madrid, 28046, Spain

  • Istituto mediterraneo trapianti - ISMETT

    RECRUITING

    Palermo, Sicily, 90127, Italy

  • Unidade Local de Saude de Coimbra Paediatric Department Avenida Afonso Romăo

    NOT_YET_RECRUITING

    Coimbra, 3000-602, Portugal

  • University Hospital Gent (UZ Gent)

    RECRUITING

    Ghent, 9000, Belgium

  • University Hospital Hamburg-Eppendorf

    RECRUITING

    Hamburg, 20246, Germany

  • University Medical Center Groningen (UMCG)

    RECRUITING

    Groningen, 9713GZ, Netherlands

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