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Liver drug maralixibat tested for Long-Term safety in rare disease patients

NCT ID NCT04168385

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 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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This study looked at the long-term safety of a drug called maralixibat in 52 people with cholestatic liver disease, including Alagille Syndrome and other rare conditions. Participants had already taken maralixibat in an earlier study. Researchers tracked side effects and how well the drug controlled itching over time.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

52 people

The number who actually took part.

Started

Jan 2020

Finished

Sep 2024

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

12 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 Subjects will need to meet all criteria below to be considered eligible for the study. 1. Provide informed consent and assent (as applicable) per the Institutional Review Board/Ethics Committee (IRB/EC). 2. Previously participated in a maralixibat study and with approval of the Medical Monitor. Previous participation is defined as: * Having completed the EOT Visit, for subjects coming from the maralixibat Phase 2 studies. * Having completed the entire duration of the study (i.e., core and extension, if applicable), for subjects coming from the maralixibat Phase 3 studies. 3. At least 1 year of age 4. Males, and females of non-childbearing potential. Males and non-pregnant, non-lactating females of childbearing potential who are sexually active must agree to use acceptable contraception during the study and 30 days following the last dose of the study medication. Females of childbearing potential must have a negative pregnancy test. 5. Caregivers (and/or age appropriate subjects) must have access to email or phone for scheduled remote visits if applicable. 6. Subject and caregiver willingness to comply with all study visits and requirements. Exclusion Criteria A subject will be excluded from the study if any of the following exclusion criteria are met: 1. Experienced an AE or SAE related to maralixibat during the lead-in protocol that led to permanent discontinuation of the subject from maralixibat. 2. Any conditions or abnormalities (including laboratory abnormalities) which, in the opinion of the Investigator or Medical Monitor may compromise the safety of the subject or interfere with the subject participating in or completing the study. 3. History of non-adherence to medical regimens, unreliability, medical condition, mental instability or cognitive impairment that, in the opinion of the Investigator or Sponsor medical monitor, could compromise the validity of informed consent, compromise the safety of the subject, or lead to non-adherence with the study protocol or inability to conduct the study procedures.

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Conditions

The condition(s) this trial relates to.

Alagille syndrome bile duct disorder biliary atresia biliary tract disorder Digestive System Diseases liver disorder progressive familial intrahepatic cholestasis

As listed by the trial registrant

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

Cholestatic liver disease

Contacts and locations

Locations

  • Baylor College of Medicine/Texas Children's Hospital

    Houston, Texas, 77030, United States

  • Birmingham Children's Hospital

    Birmingham, United Kingdom

  • Children Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States

  • Children'S Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital Westmead

    Westmead, New South Wales, 2145, Australia

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Cliniques Universitaires Saint-Luc

    Brussels, Belgium

  • Hopital Kremlin Bicetre

    Paris, 94275, France

  • Hopital Necker-Enfants Malades

    Paris, 75015, France

  • Hospital Universitario La Paz- Hospital Materno Infantil

    Madrid, 261, Spain

  • Hospital for Sick Children

    Toronto, Canada

  • Leeds Teaching Hospital NHS Trust

    Leeds, LS1 3EX, United Kingdom

  • Paediatric Liver Center, Kings College Hospital

    London, SE5 9RS, United Kingdom

  • Riley Hospital For Children

    Indianapolis, Indiana, 46202, United States

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • The Children's Memorial Health Institute

    Warsaw, 04-730, Poland

  • The Royal Children'S Hospital Melbourne

    Parkville, Victoria, 3052, Australia

More trials for these conditions

Other studies related to the condition(s) this trial covers.