Biliary Atresia Odevixibat Liver Disease (BOLD)
Research type
Research Study
Full title
A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy
IRAS ID
279864
Contact name
Lisa Karlsson
Contact email
Sponsor organisation
Albireo AB
Eudract number
2019-003807-37
ISRCTN Number
ISRCTN00000000
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
141677, IND Number; BOLD, Acronym
Duration of Study in the UK
3 years, 7 months, 8 days
Research summary
Biliary Atresia (BA) is an inborn rare disease of the liver and bile ducts (tubes) where the bile ducts outside the liver are narrow, blocked or absent. This leads to increased bile acids in the liver and worsening of liver damage. There is no approved drug treatment for BA and the only available option is a surgery called Kasai Hepatoportoenterostomy (Kasai HPE) to bypass the blocked ducts. Early surgery (within the first months after birth) is crucial for survival, without surgery liver transplantation is required or the disease is fatal. After Kasai HPE, treatment with odevixibat may be able to reduce the absorption of bile acids from the intestine back to the liver, which may lessen or delay liver damage.
This study will take place at up to 70 centres worldwide and enrol 200 infants with BA who have had Kasai HPE before they were 90 days old. Children will be assigned randomly (1 in 2 chance) to receive either study drug or placebo (dummy drug), but neither the study team nor the parent will know what they will receive (double blind study). This study includes a Screening Period of up to 3 weeks followed by a 2-year Treatment Period and a 4-week Follow-up Period. There will be a total of 11 scheduled visits during the study, after which there will be a telephone call to follow up on the child’s status. Odevixibat capsules can be opened and the pellets mixed in infant formula or breast milk and given to the child by an oral dosing syringe (or sprinkled on soft food when the child has been weaned onto solid foods). Patients will be monitored by blood and urine samples, abdominal ultrasound, and neurocognitive development testing (to measure brain function by the way the child performs tasks).
REC name
East of England - Cambridge South Research Ethics Committee
REC reference
20/EE/0142
Date of REC Opinion
11 Aug 2020
REC opinion
Further Information Favourable Opinion