Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

New drug shows promise for babies with rare liver disorders

NCT ID NCT04729751

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested the safety of a drug called maralixibat in 27 infants under 12 months old with Alagille syndrome or progressive familial intrahepatic cholestasis (PFIC), two rare liver diseases that cause bile buildup. The drug is given as an oral solution and aims to reduce bile acids and improve liver function. The main goal was to see if it is safe and tolerable in this young age group.

What this could mean

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

Active substance
maralixibat (oral solution)
What this could lead to
If it works, this could offer a way to manage cholestatic liver disease in infants, reducing itching and bile acid buildup without immediate surgery.
What could go wrong
This is a small, early-phase study focused on safety, not proof of effectiveness. The drug may not work for all types of these rare diseases, and side effects are possible.

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

27 people

The number who actually took part.

Started

Sep 2021

Finished

Dec 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

0 to 364 days

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: 1. Body weight of ≥2.5 kg 2. \<12 months of age at the baseline visit (ROW). \>31 days and \<12 months of age at the baseline visit (US). 3. Gestational age ≥36 weeks at birth. For children born with gestational age between 32 and 36 weeks, a postmenstrual age of ≥36 weeks is required. 4. Diagnosis of PFIC or ALGS Exclusion criteria: 1. Predicted complete absence of bile salt excretion pump (BSEP) function 2. History of surgical disruption of the enterohepatic circulation 3. History of liver transplant or imminent need for liver transplant 4. Decompensated cirrhosis 5. Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of drugs, including bile salt metabolism in the intestine (e.g., inflammatory bowel disease), per investigator discretion 6. Presence of other significant liver disease or any other conditions or abnormalities which, in the opinion of the investigator or medical monitor, may compromise the safety of the participant or interfere with the participant's participation in or completion of the study

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Alagille syndrome are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

Alagille syndrome bile duct disorder biliary tract disorder Cholestasis, progressive familial intrahepatic 1 liver disorder progressive familial intrahepatic cholestasis

As listed by the trial registrant

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

Alagille syndrome Cholestatic liver disease Progressive familial intrahepatic cholestasis

Contacts and locations

Locations

  • Children Hospital LA

    Los Angeles, California, 90027, United States

  • Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States

  • Cliniques Universitaires Saint-Luc

    Brussels, Belgium

  • Consultorio de Joshue David Covarrubias Esquer

    Zapopan, 45050, Mexico

  • Hopital Necker

    Paris, France

  • Hôpital Kremlin Bicêtre

    Le Kremlin-Bicêtre, France

  • Instytut Pomnik-Centrum Zdrowia Dziecka

    Warsaw, Poland

  • King's College Hospital

    London, United Kingdom

  • Medstar Georgetown University Hospital

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

  • Ochsner Hospital for Children

    New Orleans, Louisiana, 70121, United States

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • Sociedade Beneficente de Senhoras - Hospital Sírio-Libanês

    São Paulo, 01308-000, Brazil

  • Texas Children's Hospital

    Houston, Texas, 77030, United States

  • University of California - San Francisco

    San Francisco, California, 94158, United States

More trials for these conditions

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