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Hope for rare liver disease: drug aims to ease severe itching in children

NCT ID NCT04185363

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 tests the long-term safety of maralixibat, an oral medication, in children with progressive familial intrahepatic cholestasis (PFIC), a rare liver disease that causes intense itching. Participants who completed a prior study will receive maralixibat twice daily. Researchers will monitor side effects and track changes in itching severity over time.

What this could mean

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

Active substance
maralixibat
What this could lead to
If successful, this could confirm maralixibat as a safe long-term treatment to control itching and improve quality of life in children with PFIC.
What could go wrong
This is an extension study, so results depend on earlier findings. The drug may not work for everyone, and long-term side effects are still being evaluated.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

84 people

The number who actually took part.

Started

Jan 2020

Finished

Apr 2025

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

1 year to 18 years

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.

Key Inclusion Criteria: 1. Provide informed consent and assent (as applicable) per Institutional Review Board/Ethics Committee (IRB/EC) 2. Completion of study MRX-502 Exclusion Criteria: 1. Any female who is pregnant or lactating or who is planning to become pregnant 2. Administration of prohibited medication between the MRX-502 EOT visit and the MRX 503 Baseline Visit (Day 0) 3. History of non-compliance in study MRX-502, non-adherence to medical regimens, unreliability, mental instability or incompetence that could compromise the validity of informed consent or lead to non-adherence with the study protocol based on Investigator judgment 4. Experienced an adverse event (AE) or serious adverse event (SAE) related to maralixibat during the MRX-502 study that led to permanent discontinuation of the subject from maralixibat 5. Any other conditions or laboratory abnormalities that, in the opinion of the Investigator or Sponsor Medical Monitor, may compromise the safety of the subject, or interfere with the subject participating in or completing the study 6. Cognitive impairment of the subject or caregiver that would, in the opinion of the investigator, preclude appropriate understanding of study information and compliance with study procedures

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Conditions

The condition(s) this trial relates to.

bile duct disorder biliary tract disorder cholestasis Cholestasis, progressive familial intrahepatic 1 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.

Progressive familial intrahepatic cholestasis (PFIC)

Contacts and locations

Locations

  • Advent Health

    Orlando, Florida, 32803, United States

  • Azienda Ospedaliera Papa Giovanni XXIII - Unita di Pediatria

    Bergamo, 24127, Italy

  • Birmingham Children's Hospital

    Birmingham, United Kingdom

  • CHU de Toulouse - Hôpital des Enfants

    Toulouse, 31059, France

  • Children Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital at Montefiore

    The Bronx, New York, 10461, United States

  • Cincinnati Children's Hospital

    Cincinnati, Ohio, 45229, United States

  • Cliniques Universitaires Saint-Luc

    Brussels, Belgium

  • Consultario de Joshue David Covarrubias Esquer

    Zapopan, Mexico

  • Fundacion Cardioinfantil

    Bogotá, Colombia

  • Groupement Hospitalier Est Hopital, Femme Mère Enfant de Lyon

    Lyon, France

  • Hospital Italiano de Buenos Aires

    Buenos Aires, Argentina

  • Hotel Dieu de France, Alfred Naccache

    Beirut, Lebanon

  • Instytut Pomnik Centrum, Zdrowia Dziecka

    Warsaw, Poland

  • KK Women's and Children's Hospital

    Singapore, Singapore

  • King's College Hospital NHS Foundation Trust

    London, United Kingdom

  • Koc University Hospital

    Istanbul, 34010, Turkey (Türkiye)

  • Medical University of South Carolina

    Charleston, South Carolina, 29425, United States

  • Medizinische Hochschule

    Hanover, Germany

  • Medizinische Universität Wien, Universitätsklinik für Kinder- und Jugendheilkunde

    Vienna, Austria

  • Medstar Georgetown University Hospital

    Washington D.C., District of Columbia, 20007, United States

  • Ospedale Pediatrico bambino Gesu'

    Roma, Italy

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • Sociedade Beneficente de Senhoras Hospital Sírio-Libanês

    São Paulo, Brazil

  • University of Alberta - Women and Children's Health Research Institute

    Edmonton, Alberta, Canada

  • University of Texas Southwestern Medical Center

    Dallas, Texas, 75390, United States

  • University of Texas, Health Science Center San Antonio

    San Antonio, Texas, 78229, United States

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