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New drug shows promise for rare childhood liver disease

NCT ID NCT03566238

Disease control Sponsor: Albireo 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 tested a drug called odevixibat (A4250) in 62 children with progressive familial intrahepatic cholestasis (PFIC) types 1 and 2, a rare liver disease that causes severe itching and bile buildup. The drug works by blocking the reabsorption of bile acids in the gut. The goal was to see if it could lower bile acid levels and reduce itching compared to a placebo. Results could lead to a new treatment option for these children.

What this could mean

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

Active substance
odevixibat (A4250)
What this could lead to
If successful, odevixibat could become a new treatment to reduce bile acid buildup and severe itching in children with PFIC, potentially delaying or avoiding the need for liver transplant.
What could go wrong
This is a relatively small Phase 3 trial (62 children), and the drug may not work for all types of PFIC. Long-term safety and effectiveness are still being studied.

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

62 people

The number who actually took part.

Started

May 2018

Finished

Jul 2020

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

6 months 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: * A male or female participant with a clinical diagnosis of PFIC Type 1 or 2 and with a body weight above 5 kg * Participant must have clinical genetic confirmation of PFIC-1 or PFIC-2 * Participant must have elevated serum bile acid (s-BA) concentration * Participant must have history of significant pruritus and a caregiver reported observed scratching in the eDiary * Participant and/or legal guardian must sign informed consent (and assent) as appropriate. * Participants will be expected to have a consistent caregiver(s) for the duration of the study * Caregivers and age-appropriate participants (≥8 years of age) must be willing and able to use an eDiary device as required by the study Key Exclusion Criteria: * Participant with pathologic variations of the ABCB11 gene that predict complete absence of the bile salt export pump (BSEP) protein * Participant with past medical history or ongoing presence of other types of liver disease including, but not limited to, the following: 1. Biliary atresia of any kind 2. Benign recurrent intrahepatic cholestasis, indicated by any history of normal s BAs 3. Suspected or proven liver cancer or metastasis to the liver on imaging studies 4. Histopathology on liver biopsy that is suggestive of alternate non-PFIC related etiology of cholestasis * Participant with past medical history or ongoing chronic diarrhea * Any participant with suspected or confirmed cancers except for basal cell carcinoma * Participant with a past medical history of chronic kidney disease with an impaired renal function and a glomerular filtration rate \<70 mL/min/1.73 m\^2 * Participant with surgical history of disruption of the enterohepatic circulation (biliary diversion surgery) within 6 months prior to start of Screening Period * Participant has had a liver transplant or a liver transplant is planned within 6 months of randomization * Decompensated liver disease * Participant suffers from uncontrolled, recalcitrant pruritic condition other than PFIC * Participant who has been previously treated with an IBAT inhibitor whose pruritus has not responded to treatment

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Conditions

The condition(s) this trial relates to.

cholestasis progressive familial intrahepatic cholestasis type 1 progressive familial intrahepatic cholestasis type 2

As listed by the trial registrant

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

PFIC1 PFIC2

Contacts and locations

Locations

  • Akdeniz University

    Antalya, Turkey (Türkiye)

  • Astrid Lindgren Children's Hospital, Karolinska University Hospital

    Solna, Sweden

  • Azienda Ospedaliera Papa Giovanni XXIII

    Bergamo, Italy

  • Baylor College of Medicine - Texas Children's Liver Center

    Houston, Texas, 77030, United States

  • Birmingham Women's and Children's NHS Foundation Trust

    Birmingham, United Kingdom

  • British Columbia Children's Hospital

    Vancouver, Canada

  • Children's Hospital Colorado

    Denver, Colorado, 80045, United States

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States

  • Cleveland Clinic

    Cleveland, Ohio, 44195, United States

  • Cliniques Universitaires Saint-Luc

    Woluwe-Saint-Lambert, Belgium

  • Columbia University Medical Center - Presbyterian Hospital Building

    New York, New York, 10032, United States

  • Emory University School of Medicine

    Atlanta, Georgia, 30329, United States

  • Gazi University

    Ankara, Turkey (Türkiye)

  • Hacettepe University Faculty of Medicine

    Ankara, Turkey (Türkiye)

  • Hospital Necker-Enfants maladies

    Paris, France

  • Hospital Universitari Vall d'Hebron

    Barcelona, Spain

  • Hospital Universitario La Paz

    Madrid, Spain

  • Hospital de la Timone

    Marseille, France

  • Icahn School of Medicine at Mount Sinai

    New York, New York, 10029, United States

  • Inonu University Medical Faculty

    Malatya, Turkey (Türkiye)

  • Institute of Liver Studies - Kings College Hospital

    London, United Kingdom

  • Instytut Pomnik - Centrum Zdrowia Dziecka

    Warsaw, Poland

  • Istanbul University Medical Faculty

    Istanbul, Turkey (Türkiye)

  • Johns Hopkins School of Medicine

    Baltimore, Maryland, 21287, United States

  • Kinderklinik Tübingen, Universitätsklinikum Tübingen

    Tübingen, Germany

  • King Faisal Specialist Hospital & Research Centre

    Riyadh, 11211, Saudi Arabia

  • Leeds General Infirmary

    Leeds, United Kingdom

  • Medizinische Hochschule Hannover

    Hanover, Germany

  • Ospedale Regina Margherita

    Torino, Italy

  • Rambam Medical Centre

    Haifa, Israel

  • Schneider Children's Medical Center Of Israel

    Petah Tikva, Israel

  • Shaare-Zedek Mc

    Jerusalem, Israel

  • The Hospital for Sick Children

    Toronto, Canada

  • The Royal Children's Hospital

    Melbourne, Australia

  • UZ Leuven

    Leuven, Belgium

  • Uniklinikum Essen- Kinderklinik II

    Essen, Germany

  • Universitair Medisch Centrum (UMC) Utrecht

    Utrecht, Netherlands

  • Universite Paris SUD - Hopitaux Universitaires Paris-Sud - Hopital Bicetre

    Le Kremlin-Bicêtre, France

  • University Hospital Of Padova

    Padova, Italy

  • University Medical Center Groningen

    Groningen, Netherlands

  • University and Pediatric Hospital of Lyon

    Bron, France

  • University of California, San Francisco

    San Francisco, California, 94143, United States

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States