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Could this pill help kids with biliary atresia skip a liver transplant?

NCT ID NCT04336722

Disease control Sponsor: Albireo, an Ipsen Company 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jul 02, 2026 · Updated 1 time

Summary

This Phase 3 study tests odevixibat, a daily pill, in 254 children with biliary atresia who recently had Kasai surgery. The goal is to see if the drug can prevent the need for a liver transplant or death. Children take either odevixibat or a placebo for up to 2 years, and researchers track who needs a transplant or experiences serious liver events.

What this could mean

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

Active substance
odevixibat
What this could lead to
If successful, odevixibat could help children with biliary atresia avoid or delay liver transplant, improving survival without a new organ.
What could go wrong
This is a Phase 3 trial, but results are not yet available. The drug may not work better than placebo, and side effects like diarrhea or liver issues 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 3

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

Participants

254 people

The number who actually took part.

Started

Jul 2020

Finished

Jun 2026

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

Up to 111 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: * A male or female patient with a clinical diagnosis of BA * Age at Kasai HPE ≤90 days * Eligible to start study treatment within 3 weeks post-Kasai HPE Key Exclusion Criteria: * Patients with intractable ascites * Ileal resection surgery * ALT ≥10× upper limit of normal (ULN) at screening * Patients reliant only on total parenteral nutrition, or not able to take study medication orally, at randomization * Acute ascending cholangitis (patients may be randomized after resolution of acute ascending cholangitis) * Choledochal cystic disease * INR \>1.6 (the patient may be treated with Vitamin K intravenously; sample may be redrawn and if INR is ≤1.6 at resampling the patient may be randomized) * Any other conditions or abnormalities, including congenital abnormalities, major cardiac surgery, hepatic, biliary, or GI disease which, in the opinion of the Investigator or Medical Monitor, may compromise the safety of the patient, the integrity of study results, or patient compliance with study requirements * Weight \<3.5kg at randomization

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Conditions

The condition(s) this trial relates to.

biliary atresia

As listed by the trial registrant

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

Biliary atresia

Contacts and locations

Locations

  • ASST Papa Giovanni XXIII

    Bergamo, Italy

  • Akdeniz University Medical Faculty

    Antalya, Turkey (Türkiye)

  • Ann & Robert H. Lurie Children's Hospital

    Chicago, Illinois, 60611, United States

  • Bicêtre Hospital

    Le Kremlin-Bicêtre, France

  • Birmingham Women´s and Children´s Hospital

    Birmingham, United Kingdom

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • CHU Sainte-Justine

    Montreal, Canada

  • Charité - Universitätsmedizin Berlin

    Berlin, Germany

  • Children's Healthcare of Atlanta - Emory University School of Medicine

    Atlanta, Georgia, 30322, United States

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital of Fudan University

    Shanghai, China

  • Children's Mercy Hospital

    Kansas City, Missouri, 64108, United States

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010, United States

  • Cincinnati Children's Hospital

    Cincinnati, Ohio, 45229, United States

  • Cleveland Clinic

    Cleveland, Ohio, 44195, United States

  • Cliniques Universitaires Saint-Luc

    Brussels, Belgium

  • Dr von Hauner Children´s Hospital LMU

    Munich, Germany

  • Duke Central Tower

    Durham, North Carolina, 27710, United States

  • Guangzhou Women and Children's Medical Center

    Guangzhou, Guangdong, China

  • Hacettepe University Ihsan Dogramaci Childrens Hospital

    Ankara, Turkey (Türkiye)

  • Hannover Medical School

    Hanover, Germany

  • Hospital Raja Perempuan Zainab II

    Kota Bharu, Malaysia

  • Hospital Universitari Vall d'Hebron

    Barcelona, Spain

  • Hôpital Femme Mère Enfant

    Bron, France

  • ISMETT - Istituto Mediterraneo per i Trapianti e Terapie ad Alta Specializzazione

    Palermo, Italy

  • Icahn School of Medicine at Mount Sinai

    New York, New York, 10029, United States

  • Indiana University school of Medicine

    Indianapolis, Indiana, 46202, United States

  • Instytut Pomnik-Centrum Zdrowia Dziecka

    Warsaw, Poland

  • Istanbul University, Istanbul Medical Faculty

    Istanbul, Turkey (Türkiye)

  • Jeanne de Flandre Hospital

    Lille, France

  • Johns Hopkins Children's Center

    Baltimore, Maryland, 21287, United States

  • Leeds General Infirmary

    Leeds, United Kingdom

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Meyer Children´s University Hospital

    Florence, Italy

  • NYU Grossman school of Medicine

    New York, New York, 10016, United States

  • National Taiwan University Hospital

    Taipei, Taiwan

  • Necker University Hospital - Enfants malades

    Paris, France

  • Nemours/Alfred I. duPont Hospital for Children

    Wilmington, Delaware, 19803, United States

  • NewYork-Presbyterian Morgan Stanley Children's Hospital

    New York, New York, 10032, United States

  • Oregon Health & Science University

    Portland, Oregon, 97239, United States

  • Ospedale Pediatrico Bambino Gesù

    Roma, Italy

  • Rady Children's Hospital

    San Diego, California, 92123, United States

  • Regina Margherita Children´s Hospital

    Turin, Italy

  • Royal Children's Hospital

    Parkville, 3052, Australia

  • Samsung Medical Center

    Seoul, South Korea

  • Schneider Children´s Medical Center of Israel

    Petah Tikva, Israel

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • Semmelweis Egyetem I.sz Gyermekgyógyászati Klinika

    Budapest, Hungary

  • Seoul National University Children's Hospital

    Seoul, South Korea

  • Shaare Zedek Medical Center

    Jerusalem, Israel

  • Stanford Children's Health

    Palo Alto, California, 94304, United States

  • Starship Child Health

    Auckland, New Zealand

  • Taipei Veterans General Hospital

    Taipei, Taiwan

  • The Children's Hospital at Montefiore

    The Bronx, New York, 10467, United States

  • The Children´s Hospital at Westmead

    Sydney, Australia

  • The Children´s Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • The Hospital for Sick Children

    Toronto, Canada

  • UCSF Benioff Children's Hospital San Francisco

    San Francisco, California, 94158, United States

  • UPMC Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States

  • UT Southwestern Medical Center

    Dallas, Texas, 75390, United States

  • UZ Gent

    Ghent, Belgium

  • University Children´s Hospital Tuebingen

    Tübingen, Germany

  • University Hospital of Padova

    Padova, Italy

  • University Medical Center Groningen

    Groningen, Netherlands

  • University Medical Center Hamburg-Eppendorf UKE

    Hamburg, Germany

  • University of Malaya Medical Centre

    Kuala Lumpur, Malaysia

  • University of Miami

    Miami, Florida, 33146, United States

  • University of Michigan Children's Hospital

    Ann Arbor, Michigan, 48109, United States

  • University of Utah

    Salt Lake City, Utah, 84113, United States

  • Washington University in St. Louis

    St Louis, Missouri, 63110, United States

  • Women's and Children's Hospital (Women's and Children's Health Network, Incorporated.)

    North Adelaide, South Australia, Australia

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