A Study to Assess How Well and Safely Elafibranor Works in Adult Participants With Primary Sclerosing Cholangitis
This research is investigating a new medicine, elafibranor, for adults diagnosed with Primary Sclerosing Cholangitis (PSC). PSC is a rare liver condition that causes inflammation and scarring in the bile ducts, which can lead to serious liver problems over time. The study aims to find out if elafibranor is effective and safe in treating PSC, compared to a placebo (a tablet with no active medicine). About 350 people will take part, receiving either the active medicine or the placebo once a day. Researchers will regularly check participants' liver health, symptoms, and general well-being. The study will run for several years, with a follow-up period after treatment finishes, to see how the medicine impacts the disease in the long term.
At a glance
What is this study about?
This study is designed to learn more about a potential new medicine called elafibranor for a serious liver condition called Primary Sclerosing Cholangitis, or PSC. PSC is a rare disease where the small tubes in your liver, called bile ducts, become inflamed and scarred. These ducts are important because they carry bile, a fluid that helps digest food, from your liver to your gut. Over time, this scarring can damage the liver and lead to serious health issues, sometimes requiring a liver transplant. There's currently no cure, so finding new treatments is really important.
The main goal of this study is to see if elafibranor can help slow down or stop the damage to the bile ducts and improve the health of people with PSC. We also want to make sure the medicine is safe and doesn't cause too many side effects. To do this, we'll be comparing elafibranor to a 'placebo' – that's a dummy pill that looks exactly like the medicine but contains no active ingredients. This helps us understand if any changes seen are truly due to the medicine or something else.
About 350 adults with PSC will take part in this research. They will be randomly chosen to receive either elafibranor or the placebo. Participants will be carefully monitored throughout the study, with regular check-ups, blood tests, and scans. This will help doctors understand how their liver is doing, if their symptoms are improving, and how the medicine might be affecting their overall health and quality of life. The study is expected to last for several years, allowing us to see the long-term effects of the treatment.
Key takeaways
- This study is testing a new medicine called elafibranor for Primary Sclerosing Cholangitis (PSC).
- It aims to see if the medicine is safe and helps reduce liver inflammation and scarring.
- Participants will receive either elafibranor or a dummy pill (placebo) and be monitored closely.
- The study could last for several years, with regular check-ups, blood tests, and scans.
- You can withdraw from the study at any time without affecting your medical care.
Who may be eligible?
To be able to join this study, you would need to be an adult aged 18 or older with a confirmed diagnosis of Primary Sclerosing Cholangitis (PSC). Your liver disease should be 'compensated,' meaning your liver is still working reasonably well, and you must be able to follow the study's instructions and attend appointments. If you are a woman who could become pregnant, you would need to use highly effective contraception throughout the study.
There are also some reasons why you might not be able to join. For example, if you have other serious liver diseases, have had a liver transplant, or have complications from advanced liver scarring (cirrhosis). You also wouldn't be able to take part if you've had recent surgery on your bile ducts or a recent bile duct infection. Certain serious medical conditions, a history of some cancers, or taking particular medications that could interfere with the study medicine would also mean you can't participate. The study team will review all your medical information carefully to see if you are a good fit.
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 an adult aged 18 or over?
- Do you have a confirmed diagnosis of Primary Sclerosing Cholangitis (PSC)?
- Is your liver disease currently 'compensated' (still functioning reasonably well)?
- Are you able to follow study instructions and attend regular appointments?
- If you are a woman who could become pregnant, are you willing to use highly effective birth control throughout the study?
What does participation involve?
If you decide to take part, you'll first have a screening period to check if you're suitable for the study. Once accepted, you'll be randomly assigned to take either the study medicine (elafibranor) or a dummy pill (placebo) once a day. You won't know which one you're taking, and neither will your study doctor.
Throughout the study, you'll have regular visits to the clinic for check-ups. These will involve routine physical exams, blood tests, and scans to monitor your liver and general health. You'll also be asked to fill out questionnaires to tell us about your symptoms and how you're feeling. The treatment period is expected to be quite long, possibly several years, until enough health events occur among all participants to give clear results, likely around 5 years. After you finish taking the study medicine, there will be a final safety check-up about four weeks later. You are free to withdraw from the study at any time, for any reason.
Potential risks and benefits
Locations (231)
- Arizona Liver HealthVerified postcodeChandler, United States· Recruiting
- Mayo Clinic in ArizonaVerified postcodePhoenix, United States· Not yet recruiting
- Southern California Research CenterVerified postcodeCoronado, United States· Recruiting
- University of California San DiegoVerified postcodeLa Jolla, United States· Not yet recruiting
- Keck Medicine of USC - USC Healthcare Center 1Verified postcodeLos Angeles, United States· Not yet recruiting
- Cedars-Sinai Medical CenterVerified postcodeLos Angeles, United States· Not yet recruiting
- California Liver Research InstituteVerified postcodePasadena, United States· Not yet recruiting
- Stanford UniversityVerified postcodeRedwood City, United States· Not yet recruiting
- University of California Davis Medical CenterVerified postcodeSacramento, United States· Not yet recruiting
- Sutter Health Van Ness Campus Medical Office BuildingVerified postcodeSan Francisco, United States· Not yet recruiting
- University of California, San Francisco Medical CenterVerified postcodeSan Francisco, United States· Not yet recruiting
- University of Colorado Hospital Anschutz Outpatient PavilionVerified postcodeAurora, United States· Not yet recruiting
Common questions
What is Primary Sclerosing Cholangitis (PSC)?
PSC is a rare liver disease where the bile ducts inside your liver become inflamed and scarred, which can lead to liver damage over time.
What is a 'placebo'?
A placebo is a dummy pill that looks exactly like the study medicine but doesn't contain any active drug. It helps researchers understand if the real medicine is having an effect.
How long will I be in the study?
The treatment part of the study is expected to last for several years, possibly around 5 years, with a short follow-up period afterwards.
Can I stop participating in the study if I change my mind?
Yes, you are completely free to leave the study at any time, for any reason, without it affecting your regular medical care.
Will I know if I'm taking the real medicine or the dummy pill?
No, this is a 'blinded' study, meaning neither you nor your study doctor will know whether you are receiving elafibranor or the placebo.
How to find out more
Ipsen Clinical Study Enquiries
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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