Study on Obeticholic Acid for Children with Biliary Atresia After Kasai Procedure

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What is this study about?

This clinical trial is focused on studying a condition called biliary atresia, which affects the liver and bile ducts in children. The study is specifically for children who have undergone a surgical procedure known as hepatoportoenterostomy, or the Kasai procedure, which is performed to help restore bile flow. The treatment being tested in this study is a medication called obeticholic acid, also known by its code name INT-747. This medication is being compared to a placebo to evaluate its effectiveness and safety in treating biliary atresia.

The purpose of the study is to assess how well obeticholic acid works in improving clinical outcomes for children with biliary atresia who have had a successful Kasai procedure. 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 like bleeding or infections. The study will also look at how the body processes the medication and its effects on liver function and other health markers.

Participants in the study will receive either obeticholic acid or a placebo in the form of tablets. The study will be conducted over a period of time, with regular check-ups to monitor the participants’ health and any side effects. The goal is to gather information on the safety and potential benefits of obeticholic acid for children with biliary atresia, which could lead to improved treatment options in the future.

1 joining the study

Upon joining the study, the participant will be randomly assigned to receive either obeticholic acid or a placebo. This process is double-blind, meaning neither the participant nor the researchers will know which treatment is being administered.

The study is designed to assess the effects of the treatment on children with a condition called biliary atresia who have undergone a procedure known as hepatoportoenterostomy.

2 medication administration

Participants will receive the medication in the form of a tablet taken orally. The dosage will be either 0.1 mg or 1.5 mg of obeticholic acid, or a placebo tablet, depending on the group assignment.

The medication will be administered according to the study protocol, which specifies the frequency and duration of administration.

3 monitoring and assessments

Throughout the study, participants will undergo regular monitoring to assess the efficacy and safety of the treatment. This includes checking for any side effects and measuring various health indicators.

Assessments will include blood tests to measure liver function and other biomarkers, as well as physical examinations and possibly imaging tests to evaluate liver stiffness.

4 end of study participation

The study is expected to conclude by February 29, 2028. At the end of the study, participants will have a final assessment to evaluate the overall impact of the treatment.

Participants will be informed about the results of the study and any relevant findings related to their health.

Who Can Join the Study?

The study is open to both male and female children from birth up to under 18 years old. However, children under 2 years old will only be included after a safety review.
The child must have a diagnosis of non-syndromic biliary atresia, which is a liver condition.
The child must have had a successful HPE (Hepatoportoenterostomy) procedure, shown by having a total bilirubin level of less than 2 mg/dL at least 3 months after the procedure. Bilirubin is a substance made by the liver.
Female participants who can have children 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 of the study medication. This is to prevent pregnancy during the study.
Male participants who are sexually active with female partners who can have children must agree to use a condom with spermicide and another approved method of birth control from the start of the study until at least 20 days after the last dose of the study medication.
Male participants, if allowed by local rules, must not donate sperm from the start of the study until at least 20 days after the last dose of the study medication.
A parent or guardian must be willing to provide written consent for the child to participate, and the child must agree to follow the study rules if they are old enough to understand.

Who Cannot Join the Study?

Patients who have not had a successful Kasai procedure cannot participate.
Patients with a history of liver transplant are excluded.
Patients with a Pediatric End-Stage Liver Disease (PELD) score of 17 or higher, or a Model of End-Stage Liver Disease (MELD) score of 15 or higher, are not eligible. These scores are used to assess the severity of liver disease.
Patients who have been hospitalized for 24 hours or more due to new or recurring variceal bleeding cannot join. Variceal bleeding is bleeding from enlarged veins in the esophagus or stomach.
Patients with hepatic encephalopathy, which is confusion or altered level of consciousness due to liver disease, with a West Haven score of 2 or more, are excluded.
Patients with spontaneous bacterial peritonitis, a bacterial infection in the fluid in the abdomen, confirmed by a diagnostic procedure called paracentesis, cannot participate.
Patients with clinically evident ascites, which is fluid buildup in the abdomen related to high blood pressure in the liver’s blood vessels, that does not respond to diuretics and requires frequent fluid removal, are not eligible.

Where you can join this trial?

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Other Sites

Site Name	City	Country
Universitaetsklinikum Tuebingen AöR	Tuebingen	Germany
Instytut Pomnik Centrum Zdrowia Dziecka	Warsaw	Poland
Centre Hospitalier Lyon Sud	Pierre Benite	France
Ospedale Pediatrico Bambino Gesu’	Rome	Italy
Cotmorkvm Uwhcrspapsmugg Swkqwfvyt	Woluwe-Saint-Lambert	Belgium
Uipkdvuelwcyxztdxhoes Epona Atn	Essen	Germany
Ubhjukbmzkto Mjhninp Cjgtkme Gjcqmjxli	Groningen	The Netherlands
Aryboov Otgtnpojppp Pggt Gvgnrpqz Xacqc	Bergamo	Italy
Hxsolqri Vopa dkppeaop	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Belgium	Not recruiting	01.04.2024
France	Not yet recruiting	01.04.2024
Germany	Not recruiting	01.04.2024
Italy	Not recruiting	01.04.2024
Poland	Not recruiting	01.04.2024
Spain	Not recruiting	01.04.2024
The Netherlands	Not recruiting	01.04.2024

Trial locations

Investigated drugs:

Obeticholic Acid

Obeticholic Acid (OCA) is being studied to see how well it works and how safe it is for children with biliary atresia who have had a successful Kasai procedure. The trial aims to find out if OCA 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.

Biliary Atresia, Post-hepatoportoenterostomy – Biliary atresia is a rare liver disease that affects infants, where the bile ducts become blocked or absent. This condition leads to bile buildup in the liver, causing damage and scarring. Post-hepatoportoenterostomy refers to the state after a surgical procedure called the Kasai procedure, which aims to restore bile flow from the liver to the intestine. Despite the surgery, many children may still experience liver damage over time. The disease can progress to liver failure, necessitating further medical interventions. Monitoring and managing symptoms are crucial to address complications that may arise.

Trial ID:

2023-503926-37-00

Protocol code:

747-308

NCT ID:

NCT06121375

Trial Phase:

Therapeutic use (Phase IV)

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Study on Obeticholic Acid for Children with Biliary Atresia After Kasai Procedure

What is this study about?

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