Active not recruitingPHASE3INTERVENTIONAL

Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)

This research is looking at a new medicine called Odevixibat for children diagnosed with a serious liver condition known as Biliary Atresia. These children have already had an operation called a Kasai procedure. The study wants to find out if Odevixibat is both safe to use and effective in helping these children, compared to a placebo (a dummy medicine). Doctors and researchers are carefully checking if it improves health outcomes. This is a "Phase 3" study, which means it's one of the final steps before a medicine might be made widely available. It's a double-blind study, meaning neither the patient's family nor the doctors will know who is getting the real medicine and who is getting the placebo, to keep the results fair and unbiased.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Albireo, an Ipsen Company

Enrolment target

254

Start

08 Jul 2020

Estimated completion

22 Jun 2026

Biliary Atresia

What is this study about?

Biliary Atresia is a rare but serious liver condition that affects new-born babies. It means that the ducts (small tubes) in the liver that carry a fluid called bile to the gut are blocked or haven't formed properly. Bile helps the body digest food, and if it builds up in the liver, it can cause severe damage. Most babies with Biliary Atresia need an operation called a Kasai procedure, where the surgeon connects part of the small intestine directly to the liver to help drain the bile. While this operation can help, it doesn't always solve the problem completely, and children often need further treatment or even a liver transplant later on.

This study is investigating a new medicine called Odevixibat. Researchers believe this medicine might help improve the flow of bile or reduce its build-up in the liver after a Kasai procedure. The main goal is to see if Odevixibat can improve the health of children with Biliary Atresia and potentially reduce the need for more complex treatments in the future. By comparing it to a placebo (a medicine with no active ingredients), the researchers can get a clear picture of how effective and safe Odevixibat really is.

This kind of study, called a Phase 3 trial, is very important. It involves a larger number of patients and gives researchers detailed information on whether the medicine works well enough and is safe enough to be considered for wider use. The results from this study will help doctors understand how best to care for children with Biliary Atresia after their Kasai operation.

Key takeaways

This study is testing a new medicine for babies with Biliary Atresia.
It's for babies who have already had a Kasai operation.
The study compares the new medicine (Odevixibat) to a dummy medicine (placebo).
It's a Phase 3 study to check safety and effectiveness.
Regular check-ups and monitoring will be part of participation.

Who may be eligible?

This study is designed for babies diagnosed with Biliary Atresia. To take part, your baby must have had their Kasai operation when they were 90 days old or younger, and they need to be able to start the study medicine within three weeks after that operation.

There are several reasons why a baby might not be able to join the study. For example, if they have severe fluid build-up in their tummy (intractable ascites), or if they've had surgery to remove part of their small intestine. Babies with very high liver enzyme levels (ALT) or those who can only be fed through a drip, or can't take medicine by mouth, also cannot participate. If your baby has an infection of the bile ducts (cholangitis) or another specific liver condition called choledochal cystic disease, they wouldn't be suitable. Also, if they have other serious health problems that the doctors think would make the study unsafe or difficult to interpret, they might not be able to join. Finally, babies weighing less than 3.5kg at the time of joining cannot take part.

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.

Has your child been diagnosed with Biliary Atresia?
Did your child have their Kasai operation when they were 90 days old or younger?
Is your child able to start study treatment within 3 weeks of their Kasai operation?
Does your child currently weigh at least 3.5kg?
Can your child take medicine by mouth (not only through an IV drip)?
Does your child *not* have severe fluid in their tummy or certain other serious health issues?

Answer every question to see your result.

What does participation involve?

If your child takes part in this study, they will receive either the study medicine, Odevixibat, or a placebo (a dummy medicine). Neither you nor your doctor will know which one they are receiving. Your child will have regular check-ups, blood tests, and other assessments to monitor their health, see how the medicine is working, and check for any side effects. These visits will be scheduled regularly over the study period. The doctors and nurses will explain exactly what each visit involves and how long it will take. There will also be follow-up appointments after your child finishes taking the study medicine to ensure their well-being.

Potential risks and benefits

Taking part in any medical study carries potential benefits and risks. Your child might benefit from receiving a new medicine that could improve their condition, or they might receive the placebo. However, there's no guarantee the medicine will work, and it might have side effects, which the study will carefully monitor. The doctors will explain all known potential risks before you decide. You are free to withdraw your child from the study at any time, for any reason, without it affecting their ongoing medical care.

Locations (72)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Children's Hospital Los Angeles
Verified postcode
Los Angeles, United States
Stanford Children's Health
Verified postcode
Palo Alto, United States
Rady Children's Hospital
Verified postcode
San Diego, United States
UCSF Benioff Children's Hospital San Francisco
Verified postcode
San Francisco, United States
Children's Hospital Colorado
Verified postcode
Aurora, United States
Nemours/Alfred I. duPont Hospital for Children
Verified postcode
Wilmington, United States
Children's National Medical Center
Verified postcode
Washington D.C., United States
University of Miami
Verified postcode
Miami, United States
Children's Healthcare of Atlanta - Emory University School of Medicine
Verified postcode
Atlanta, United States
Ann & Robert H. Lurie Children's Hospital
Verified postcode
Chicago, United States
Indiana University school of Medicine
Verified postcode
Indianapolis, United States
Johns Hopkins Children's Center
Verified postcode
Baltimore, United States

Common questions

What is Biliary Atresia?

Biliary Atresia is a rare liver condition in babies where bile ducts are blocked, stopping bile from reaching the gut and causing liver damage.

What is a Kasai procedure?

It's an operation for Biliary Atresia where a surgeon connects the small intestine directly to the liver to help bile drain.

What is "double-blind"?

It means neither you, your child's family, nor the doctors will know if your child is getting the real medicine or a dummy medicine (placebo).

What is Odevixibat?

It's the new medicine being tested in this study, designed to help manage Biliary Atresia after a Kasai procedure.

Can I take my child out of the study?

Yes, you can choose to withdraw your child from the study at any time, for any reason, and it won't affect their care.