Study on Long-term Effects and Safety of Odevixibat in Children with Biliary Atresia

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What is this study about?

This clinical trial is focused on studying the long-term effects of a medication called odevixibat (also known by its code name A4250) in children with a liver disease known as biliary atresia. Biliary atresia is a condition that affects the bile ducts, which are tubes that carry bile from the liver to the gallbladder. In this disease, the bile ducts are blocked or absent, leading to liver damage. The purpose of the study is to evaluate how well odevixibat works over a long period and to ensure it is safe for children with this condition.

Participants in the study will take odevixibat in the form of a capsule, which is taken by mouth. The study will follow the children over a period of time to monitor their liver health and overall growth. The main focus will be on how long the children can maintain their liver function without needing a liver transplant or experiencing severe liver problems. The study will also look at changes in certain blood tests that indicate liver health, as well as the children’s growth and development compared to standard growth charts.

The study is an extension of a previous trial, meaning it will continue to observe children who have already been taking odevixibat to see if the benefits are sustained over time. The researchers will collect data at various points throughout the study to assess the medication’s impact on liver health and overall well-being. This information will help determine if odevixibat is a viable long-term treatment option for children with biliary atresia.

1 joining the study

Upon joining the study, you will be required to have completed the 104-week treatment period of the previous study, A4250-011. Your parent or legal guardian must provide signed informed consent for your participation.

2 medication administration

You will be taking a medication called odevixibat, which is also known as A4250. This medication is administered orally in the form of a capsule. The specific dosage and frequency will be determined by the study team based on your individual needs.

3 treatment duration

The treatment period for this study is planned to last up to 104 weeks. During this time, you will continue to take the medication as directed by the study team.

4 regular assessments

Throughout the study, you will undergo regular assessments to monitor your health and the effects of the medication. These assessments will include measuring changes in liver function, growth, and other health indicators at specified intervals: Weeks 4, 13, 26, 39, 52, 78, and 104.

5 monitoring outcomes

The primary outcome being monitored is the time from the first dose of the study treatment to the first occurrence of a liver transplant or death. Secondary outcomes include changes in liver function scores, growth measurements, and other health indicators over the course of the study.

6 completion of study

At the end of the 104-week treatment period, your participation in the study will be complete. The study team will review all collected data to evaluate the long-term efficacy and safety of the medication.

Who Can Join the Study?

  • The patient must have completed the 104-week Treatment Period of Study A4250-011.
  • The patient’s parent(s) or legal guardian must have signed an informed consent form. This means they agree to let the patient participate in the study after understanding all the details.
  • The patient can be of any gender, as both male and female participants are allowed.
  • The study is open to children, as the age range includes young participants.

Who Cannot Join the Study?

  • Patients who have not completed the previous study called A4250-011 (BOLD).

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Medizinische Hochschule Hannover Hanover Germany
Centre Hospitalier Universitaire De Lille Lille France
Hopital Necker Enfants Malades Paris France

Other Sites

Site Name City Country Status
Azienda Ospedaliera di Padova Padua Italy
Instytut Pomnik Centrum Zdrowia Dziecka Warsaw Poland
Semmelweis University Budapest Hungary
Universitaetsklinikum Tuebingen AöR Tuebingen Germany
Hospices Civils De Lyon Lyon France
Azienda Ospedaliera Universitaria Meyer IRCCS Florence Italy
Universitair Ziekenhuis Gent Gent Belgium
Deutsches Herzzentrum Berlin Berlin Germany
Aakezgkipq Pkwcrtng Hcdalroi Di Pagld Le Kremlin-Bicetre France
Avnooms Olhbrfevczu Udcwrqjvajshh Ckoeysizyhah Dpsmh Sother E Djqbc Sbjqbko Dt Tttbgf Turin Italy
Ulxcmafbts Mozrugn Ctqeoa Huyhjhocokccydakv Hamburg Germany
Ahbdbor Owgrmhixvjf Pjzq Gmwqpvpp Xpcoe Bergamo Italy
Uhhdiycocxfq Muassfl Cfjlulw Ghfiqeybw Groningen The Netherlands
Hcpoolyy Vftc ddhfmsat Barcelona Spain

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Belgium Belgium
Not recruiting
13.12.2022
France France
Recruiting
13.12.2022
Germany Germany
Recruiting
13.12.2022
Hungary Hungary
Recruiting
13.12.2022
Italy Italy
Recruiting
13.12.2022
Poland Poland
Recruiting
13.12.2022
Spain Spain
Recruiting
13.12.2022
The Netherlands The Netherlands
Recruiting
13.12.2022

Trial locations

Investigated drugs:

Odevixibat is a medication being studied for its potential to help children with a liver condition called biliary atresia. This condition affects the bile ducts, which are tubes that carry bile from the liver to the gallbladder and small intestine. Bile is important for digestion and removing waste from the body. In biliary atresia, these ducts are blocked or damaged, leading to liver problems. Odevixibat works by reducing the amount of bile acids in the liver and intestines, which may help improve liver function and increase the chances of the liver working well for a longer time. This study is looking at how safe and effective odevixibat is when used over a long period in children who have already participated in a previous study with this medication.

Biliary Atresia – Biliary atresia is a condition in infants where the bile ducts inside or outside the liver do not develop normally. This leads to a blockage in the flow of bile, a digestive fluid produced by the liver. As a result, bile becomes trapped in the liver, causing damage and scarring of liver cells. Over time, this can lead to liver dysfunction and cirrhosis. The disease typically presents with symptoms such as jaundice, dark urine, and pale stools. If untreated, the condition can progress, affecting the liver’s ability to function properly.

Trial ID:
2022-501090-39-00
Protocol code:
A4250-016
Trial Phase:
Therapeutic confirmatory (Phase III)

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