An Open-label Study to Evaluate Efficacy and Safety of Odevixibat in Children with Biliary Atresia
Research type
Research Study
Full title
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia (BOLD-EXT)
IRAS ID
1006307
Contact name
Deborah Westwood
Contact email
Sponsor organisation
Albireo AB
Clinicaltrials.gov Identifier
Research summary
This is an Open-label Extension Study of an investigational drug (also known as the “study drug”) called odevixibat as a possible treatment for biliary atresia (BA). An open-label study is a type of study in which both participant and study doctor will know which medication is being given. All children in the open-label study will receive the study drug, odevixibat, even if they previously received placebo in the A4250-011 study (BOLD). An investigational drug is one that has not been approved for use by health authorities.
The study drug is believed to reduce the absorption of bile acids from the intestine back to the liver, thus lowering bile acid levels in the blood and liver, which may lessen or delay liver damage. Odevixibat has so far been tested in healthy volunteers, in a study in children (aged 6 months old or older) with cholestatic liver diseases, and in the BOLD study.
The main purpose of this study is to learn how safe and effective the study drug is long-term.
REC name
West of Scotland REC 1
REC reference
23/WS/0050
Date of REC Opinion
30 Aug 2023
REC opinion
Further Information Favourable Opinion