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

NCT ID NCT04336722

Completed Disease control Sponsor: Albireo, an Ipsen Company Source: ClinicalTrials.gov ↗

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.

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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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