A Randomized, Double-blind, Placebo-controlled, Phase 2/3 Study to Assess the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Obeticholic Acid Compared to Placebo in Pediatric Subjects with Biliary Atresia, Post-hepatoportoenterostomy
This research study, called a clinical trial, is looking at a new medicine called obeticholic acid (OCA) for children and young adults who have a rare liver disease called biliary atresia. These patients have also had a surgery called a hepatoportoenterostomy. The study aims to find out if OCA is safe and effective in slowing the progression of their liver disease. We are comparing OCA to a placebo (a dummy pill) to see if it can help prevent serious events like liver transplant, severe liver problems, or hospital stays due to complications. The study will also help us understand how the medicine works in the body.
At a glance
What is this study about?
This study is about a medicine called obeticholic acid (which we'll call OCA) for children and young adults who have a rare liver condition called biliary atresia. Biliary atresia is a disease found in newborn babies where the tubes that carry bile (a digestive fluid) out of the liver don't form properly. This causes bile to build up in the liver, which can lead to serious liver damage.
Many children with biliary atresia have an operation called a hepatoportoenterostomy (also known as the Kasai procedure) to try and create a way for the bile to drain. While this surgery can help, many still face ongoing liver problems. This study wants to see if OCA can help those who have had this surgery. We're hoping it can slow down the liver disease and improve their health over time.
Researchers will be looking at important health milestones, often called 'events,' to see how well the medicine is working. These events include things like needing a liver transplant, developing serious liver complications, or being admitted to the hospital because of liver problems. By comparing the effects of OCA to a placebo (a pill that looks the same but contains no medicine), the researchers can understand if OCA is truly beneficial for these patients.
Key takeaways
- This study is testing a new medicine (OCA) for children and young adults with biliary atresia after surgery.
- It aims to see if OCA can slow down liver disease and prevent serious health problems.
- Participants will receive either OCA or a placebo, and will have regular health checks.
- The study will look at how safe the medicine is and how well it works.
- You can choose to leave the study at any time if you wish.
Who may be eligible?
This study is looking for children and young adults aged 18 years and younger who have biliary atresia and have already had a special surgery for their condition. Both boys and girls can take part.
To join, a patient must meet certain health requirements that the study doctors will check carefully. These include specific details about their liver health, other medical conditions, and medications they might be taking. The study team will review all these factors to make sure that taking part is safe and appropriate for each individual.
The most important thing is that the patient's doctor believes that this study is a good option for them and that they meet all of the study's specific health criteria. This helps make sure the study gathers the best information possible and that participants are kept safe.
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.
- Are you 18 years old or younger?
- Do you have biliary atresia?
- Have you had a hepatoportoenterostomy (Kasai procedure)?
- Are you able to attend regular clinic visits and follow study instructions?
- Your doctor thinks this study might be a good option for you.
What does participation involve?
If you decide to take part in this study, you would be randomly chosen to receive either the study medicine (OCA) or a placebo (a dummy pill). Neither you nor your doctor would know which one you are receiving. You would take this medicine as directed by the study team.
You would have regular visits to the hospital or clinic. During these visits, the study team would perform various health checks, which might include blood tests, physical examinations, and possibly scans to look at your liver. These checks help the doctors monitor your health, see how the medicine is working, and watch for any side effects.
The study would last for a period of time, and you would continue to take the study medicine and attend these follow-up appointments. The exact number of visits and the total length of your participation would be explained in detail by the study team.
Potential risks and benefits
Locations (7)
- —UnverifiedBelgium
- —UnverifiedGermany
- —UnverifiedNetherlands
- —UnverifiedFrance
- —UnverifiedItaly
- —UnverifiedSpain
- —UnverifiedPoland
Common questions
What is biliary atresia?
It's a rare liver disease in babies where tubes carrying bile out of the liver don't form properly, leading to liver damage.
What is a hepatoportoenterostomy?
It's a surgery for biliary atresia to help bile drain from the liver, also known as the Kasai procedure.
What does 'placebo' mean?
A placebo is a dummy pill that looks like the study medicine but doesn't contain any active drug. It helps compare the new medicine's effects.
Will I know if I'm getting the real medicine or the placebo?
No, this is a 'blinded' study, meaning neither you nor your doctor will know until the study is over.
Can I leave the study if I want to?
Yes, you can leave the study at any time, and it won't affect your regular medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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