In short
There are currently 2 ongoing clinical trials investigating treatments for congenital absence of bile ducts, also known as biliary atresia. These studies are testing medications called odevixibat and obeticholic acid in children who have undergone or are living with this rare liver condition. Trials are taking place across several European countries including Belgium, France, Germany, Hungary, Italy, the Netherlands, Poland, and Spain.
Clinical trial locations
- Belgium
- France
- Germany
- Hungary
- Italy
- Netherlands
- Poland
- Spain
Study on Long-term Effects and Safety of Odevixibat in Children with Biliary Atresia
This study is examining the long-term effects and safety of a medication called odevixibat in children with biliary atresia, a condition where the bile ducts that carry bile from the liver are blocked or absent. The trial is an extension study, meaning it continues to follow children who have already participated in a previous trial with this medication.
Who can participate:
- Children of any gender who have completed the full 104-week treatment period of the previous study called A4250-011
- Parent or legal guardian must provide signed consent for participation
Who cannot participate:
- Patients who have not completed the previous A4250-011 study
What the study involves:
The study aims to evaluate how well odevixibat works over a long period and to ensure it is safe for children with this condition. Participants will take odevixibat as an oral capsule over a treatment period lasting up to 104 weeks. The study will monitor several important outcomes including how long children can maintain their liver function without needing a liver transplant or experiencing severe liver problems. Regular assessments will occur at weeks 4, 13, 26, 39, 52, 78, and 104 to monitor liver health, growth, and development. Blood tests will measure changes in liver function, and growth measurements will be compared to standard growth charts.
About the medication:
Odevixibat works by reducing the amount of bile acids in the liver and intestines. It does this by blocking the reabsorption of bile acids in the intestines, which helps decrease the buildup of these acids in the liver. This may help improve liver function and increase the chances of the liver working well for a longer time. The medication is classified as an ileal bile acid transporter inhibitor.
Study on Obeticholic Acid for Children with Biliary Atresia After Kasai Procedure
This trial is studying a medication called obeticholic acid in children with biliary atresia who have undergone a surgical procedure known as the Kasai procedure, or hepatoportoenterostomy. This surgery is performed to help restore bile flow from the liver. The medication is being compared to a placebo to evaluate its effectiveness and safety.
Who can participate:
- Male and female children from birth up to under 18 years old (children under 2 years will only be included after a safety review)
- Diagnosis of non-syndromic biliary atresia
- Successful Kasai procedure demonstrated by a total bilirubin level of less than 2 mg/dL at least 3 months after the procedure
- Female participants who can become pregnant must use at least one highly effective method of birth control from the start of the study until at least 20 days after the last dose
- Male participants who are sexually active must agree to use contraception during the study period
- Parent or guardian must provide written consent, and the child must agree if old enough to understand
Who cannot participate:
- Patients who have not had a successful Kasai procedure
- Patients with a history of liver transplant
- Patients with severe liver disease as indicated by high PELD scores (17 or higher) or MELD scores (15 or higher)
- Patients hospitalized for 24 hours or more due to bleeding from enlarged veins in the esophagus or stomach
- Patients with confusion or altered consciousness due to liver disease
- Patients with bacterial infection in the abdominal fluid
- Patients with significant fluid buildup in the abdomen that doesn't respond to diuretics
What the study involves:
This is a double-blind study, meaning neither the participants nor the researchers will know who is receiving the active medication or placebo. Participants will receive either obeticholic acid (in doses of 0.1 mg or 1.5 mg) or a placebo tablet taken orally. The study will monitor various health events such as the need for a liver transplant, changes in liver disease severity, and hospitalizations due to complications. Regular monitoring will include blood tests to measure liver function and other health indicators, physical examinations, and possibly imaging tests to evaluate liver stiffness. The study is expected to conclude by February 2028.
About the medication:
Obeticholic acid is being studied to see if it can improve clinical outcomes by delaying or preventing serious liver-related events such as liver transplant, severe liver disease, or hospitalization due to liver complications. It works by activating a specific receptor in the liver called the farnesoid X receptor, which helps to reduce inflammation and scarring in the liver. This medication is classified as a farnesoid X receptor agonist.
Summary
Both clinical trials are focused on improving outcomes for children with biliary atresia, a rare condition affecting the bile ducts. The trials are testing two different medications that work through different mechanisms: odevixibat reduces bile acid reabsorption in the intestines, while obeticholic acid activates specific liver receptors to reduce inflammation.
Geographically, these trials show widespread participation across Europe, with both studies being conducted in Belgium, France, Germany, Italy, the Netherlands, Poland, and Spain. Hungary also participates in the odevixibat study. This broad distribution reflects international collaboration in researching treatments for this rare childhood liver condition.
The odevixibat study is an extension trial focusing on long-term safety and effectiveness in children who have already been taking the medication, while the obeticholic acid study is evaluating a new treatment option specifically for children who have had the Kasai surgical procedure. Both studies emphasize careful monitoring of liver function and aim to delay or prevent the need for liver transplantation while improving quality of life for affected children.
