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 (BOLD)
Biliary atresia is a rare liver disease in babies where bile ducts are blocked or missing. Most babies undergo a Kasai operation to help bile drain into the bowel. Even after surgery, many children continue to experience liver scarring and may eventually need a liver transplant. This phase 3 clinical study, named BOLD, is testing whether an investigational medicine called odevixibat (A4250) can help keep the liver healthy. It compares odevixibat to a dummy treatment (placebo) over two years (104 weeks). Children in the trial take the study medicine by mouth once a day. Doctors will closely check liver health, blood tests (including bilirubin and bile acid levels), and overall growth. The main aim is to discover whether odevixibat safely delays or reduces the need for a liver transplant.
At a glance
What is this study about?
Biliary atresia is a rare and serious liver condition that affects newborn babies. In a healthy body, tiny tubes called bile ducts carry bile—a fluid that helps digest food—from the liver into the gut. In children with biliary atresia, these ducts become inflamed and blocked. As a result, bile builds up in the liver, leading to jaundice, inflammation, and progressive scarring.
The usual first step in treatment is an operation known as a Kasai procedure. This surgery rebuilds a connection between the liver and the small intestine to restore bile drainage. Although a Kasai operation can improve health, it is rarely a complete cure. Many children still face ongoing liver damage and may require a liver transplant later in childhood.
This clinical study is evaluating a daily medicine called odevixibat. It works by blocking the recycling of bile acids from the gut back to the liver, which helps clear harmful substances. Researchers hope this action will protect liver cells, lower jaundice levels, and allow children to keep their own liver for longer.
Because this is a Phase 3 study, researchers will compare odevixibat directly with an inactive dummy pill (placebo). This helps doctors gather clear, trustworthy evidence on how safe the medicine is and whether it truly improves the long-term health of children living with biliary atresia.
Key takeaways
- Tests whether odevixibat can delay or prevent the need for a liver transplant in children with biliary atresia.
- Open to children who have already had a Kasai operation.
- Compares the active drug to an inactive dummy capsule (placebo) over two years.
- Capsules can be opened and mixed into food for babies and young children.
- Participation is completely voluntary and will not change your child's standard NHS treatment.
Who may be eligible?
This study is designed specifically for children diagnosed with biliary atresia who have already undergone a Kasai operation. Both boys and girls can take part.
To join, children must meet certain medical requirements regarding their current liver function and general health. Children who are already on an urgent waiting list for an immediate liver transplant, or who have other severe medical complications, may not be able to participate.
The hospital care team will carry out detailed health checks, including physical exams and blood tests, to make sure taking part is appropriate and safe for each child.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- My child has a confirmed diagnosis of biliary atresia.
- My child has already had a Kasai operation.
- My child is able to take daily oral medicine (either as a capsule or sprinkled on food).
- We are able to attend regular hospital check-ups and blood tests over a two-year period.
- My child is not currently listed for an immediate, urgent liver transplant.
What does participation involve?
Taking part in the study lasts for 104 weeks (about two years). Children are assigned by chance (randomly) to take either odevixibat or a matching placebo capsule once every day. To keep results fair, neither parents nor the medical team will know which treatment the child is receiving.
The medicine is supplied as capsules filled with tiny pellets. For infants and toddlers who cannot swallow whole capsules, the capsules can be opened and the pellets mixed with soft food or drink. Throughout the two years, families will visit the hospital clinic regularly. These visits will include physical check-ups, tummy ultrasound scans, and blood tests to measure bile acids, bilirubin, and vitamin levels. The study team will monitor each child's health and development closely.
Potential risks and benefits
Locations (8)
- —UnverifiedHungary
- —UnverifiedBelgium
- —UnverifiedFrance
- —UnverifiedSpain
- —UnverifiedPoland
- —UnverifiedGermany
- —UnverifiedItaly
- —UnverifiedNetherlands
Common questions
What is a placebo and why is it used?
A placebo looks and tastes just like the active medicine but contains no active drug. Comparing odevixibat to a placebo helps doctors see whether changes in health are truly caused by the new treatment.
How do young children take the capsules?
The capsules are filled with tiny coated pellets. If your child cannot swallow capsules, you can open them and sprinkle the contents onto a small spoonful of soft food or liquid.
Can my child keep taking their usual liver medicines?
Most routine medications, such as fat-soluble vitamins and antibiotics, can continue. The study doctor will review all of your child's medicines to make sure they are safe to use together.
What happens if my child's liver gets worse during the study?
Your child's health is always the top priority. If blood tests or scans show that their liver disease is worsening, the medical team will immediately provide the necessary care, which may include stopping the trial medicine.
Do we have to pay to take part?
No. The study medication, clinic visits, and all specialised tests are provided free of charge. Travel and parking costs for hospital appointments are typically refunded.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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