New drug may help kids with rare liver disease avoid transplant

NCT ID NCT05426733

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

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests a drug called odevixibat in 180 children with biliary atresia, a rare liver disease. The goal is to see if the drug helps children stay alive without needing a liver transplant over the long term. Children who completed a previous study can join this open-label extension, meaning everyone gets the drug.

What this could mean

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

Active substance

odevixibat (a drug that blocks bile acid absorption in the gut)

What this could lead to

If it works, this could help children with biliary atresia avoid needing a liver transplant for longer.

What could go wrong

This is an extension study, so it builds on earlier results. The drug may not prevent transplant in all children, and side effects are possible.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

Get updates

Get notified about this study

Our safety recommendation!

By submitting, you agree to our Terms of use

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

Show contact details

Locations

ASST Papa Giovanni XXIII

Bergamo, Italy
Akdeniz University Medical Faculty

Antalya, Turkey (Türkiye)
Azienda Ospedaliera di Padova

Padova, Italy
Bicêtre Hospital

Le Kremlin-Bicêtre, France
Boston Children's Hospital

Boston, Massachusetts, 02115, United States
CHU Sainte-Justine

Montreal, Canada
Charité - Universitätsmedizin Berlin

Berlin, Germany
Children's Healhcare of Atlanta- Emory University school of medicine

Atlanta, Georgia, 30322, United States
Children's Hospital Colorado

Aurora, Colorado, 80045, United States
Children's Hospital of Fudan University

Shanghai, China
Children's Mercy Hospital and Clinics

Kansas City, Missouri, 64108, United States
Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States
Columbia University Medical Center New York Presbyterian Morgan Stanley-Komansky Childrens Hospital

New York, New York, 10032, United States
Guangzhou Women And Childrens Medical Center

Guangdong, China
Hacettepe İhsan Doğramacı Children's Hospital

Ankara, Turkey (Türkiye)
Hassenfeld Children's Hospital at NYU Langone

New York, New York, 10016, United States
Hospital Raja Perempuan Zainab II

Kota Bharu, Malaysia
Hospital Universitari Vall d'Hebron

Barcelona, Spain
Indiana University school of Medicine

Indianapolis, Indiana, 46202, United States
Instytut Pomnik-Centrum Zdrowia Dziecka

Warsaw, Poland
Istanbul University Istanbul Medical Faculty Hospital

Istanbul, Turkey (Türkiye)
Royal Children's Hospital

Parkville, 3052, Australia
Seattle Children's Hospital

Seattle, Washington, 98105, United States
Seoul National University Childrens Hospital

Seoul, South Korea
Starship Child Health, Te Whatu Ora - Health New Zealand

Auckland, 1023, New Zealand
The Childrens Hospital at Montefiore Albert Einstein School of Medicine

The Bronx, New York, 10467, United States
The Hospital for Sick Children

Toronto, Canada
University Children´s Hospital Tuebingen

Tübingen, Germany
University Malaya Medical Centre

Kuala Lumpur, 59100, Malaysia
University Medical Center Groningen

Groningen, Netherlands