Active not recruitingPHASE2INTERVENTIONAL

A Study to Assess Safety and Effectiveness of Elafibranor in Adult Participants With Primary Sclerosing Cholangitis.

This study is investigating a new medication, elafibranor, for adults who have a rare liver disease called Primary Sclerosing Cholangitis (PSC). PSC causes damage to the small tubes in the liver that carry a fluid called bile. Over time, this damage can worsen the liver condition. The study aims to understand if elafibranor is safe to use and what side effects it might have. We will also carefully check if the medicine helps improve aspects of PSC, using blood tests and other assessments. Some participants will receive elafibranor, while others will get a placebo (a dummy treatment) to help us compare the effects fairly.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Ipsen

Enrolment target

Start

29 Dec 2022

Estimated completion

28 Aug 2026

Primary Sclerosing Cholangitis

What is this study about?

This research study is about a new medicine called elafibranor for a liver disease known as Primary Sclerosing Cholangitis, or PSC. PSC is a rare condition where the small tubes inside your liver, called bile ducts, get damaged and scarred. These bile ducts are important because they carry bile, a fluid that helps digest food, from your liver. When they get damaged, bile can build up in the liver, which can cause more damage over time and make the disease worse.

The main goal of this study is to find out if elafibranor is safe for people with PSC and what its side effects might be. We also want to see if this medicine can help improve the condition by looking at certain blood tests and other checks related to PSC activity. To do this, some people will receive elafibranor, while others will get a 'placebo', which looks exactly like the study medicine but contains no active drug. This helps us understand if any changes are truly due to the medicine or something else.

Taking part in a study like this helps doctors learn more about diseases like PSC and develop new treatments. It's an important step in finding better ways to manage the condition and improve the lives of people living with it.

Key takeaways

This study is for adults with a rare liver condition called Primary Sclerosing Cholangitis (PSC).
It's testing a new medicine, elafibranor, to see if it's safe and helpful.
Some participants will receive the study medicine, others a placebo (dummy treatment).
You'll have regular hospital or clinic visits for blood tests and checks.
The study aims to improve understanding and treatment options for PSC.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

This study is looking for adults aged between 18 and 75 who have been diagnosed with Primary Sclerosing Cholangitis (PSC). To join, you should have clear medical evidence of PSC from scans and blood tests, and your PSC should not be due to other known causes.

There are also specific requirements for your blood test results, particularly for liver health markers like Alkaline Phosphatase (ALP) and total bilirubin, which need to be within certain ranges. If you are taking medication for PSC, like ursodeoxycholic acid (UDCA), it needs to have been at a steady dose for at least six months. If you recently stopped UDCA, you'd need to have been off it for at least three months before the study starts.

If you also have Inflammatory Bowel Disease (IBD), such as Crohn's disease or ulcerative colitis, it needs to be well-controlled and stable on your current treatment for at least three months. You would also need to have had a colonoscopy within the past year with no signs of serious changes. There are also considerations for other medications you might be taking to manage PSC symptoms like itching.

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 between 18 and 75 years old?
Have you been diagnosed with Primary Sclerosing Cholangitis (PSC)?
Do your recent blood tests for liver function meet certain requirements?
If you take UDCA, has your dose been stable for at least 6 months (or off for 3 months)?
If you have Inflammatory Bowel Disease (IBD), is it well-controlled?
Have you had a colonoscopy within the last year with no signs of serious changes (if you have IBD)?

Answer every question to see your result.

What does participation involve?

If you join this study, you'll be assigned to take either one of two doses of elafibranor (80mg or 120mg) or a placebo (a dummy treatment) in pill form. You will take this medication daily. You'll have regular visits to the hospital or clinic for check-ups, blood tests, and other assessments to monitor your health and how you're responding to the treatment. These tests will include checking your liver function and overall well-being. The study will involve a main treatment period, followed by a period where your health continues to be monitored. The total duration of your participation, including follow-up, will be explained in detail by the study team.

Potential risks and benefits

Taking part in a research study has potential benefits and risks. You might not directly benefit from taking elafibranor, as it's an investigational medicine, but the information gained from the study could help future patients with PSC. Potential risks include side effects from the study drug, which will be carefully monitored. The study team will explain all known potential side effects and what to look out for. You will also have additional blood tests and check-ups. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (62)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Om Research LLC
Verified postcode
Lancaster, United States
Cedars-Sinai Medical Center
Verified postcode
Los Angeles, United States
University of California, Davis
Verified postcode
Sacramento, United States
Sutter Health Van Ness Campus Medical Office Building
Verified postcode
San Francisco, United States
Peak Gastroenterology Associates
Verified postcode
Colorado Springs, United States
South Denver Gastroenterology,P.C.
Verified postcode
Englewood, United States
Rocky Mountain Gastroenterology (RMG)
Verified postcode
Littleton, United States
Yale University School Of Medicine - Yale Center For Clinical Investigation
Verified postcode
New Haven, United States
Schiff Center for Liver Diseases - University of Miami
Verified postcode
Miami, United States
Covenant Research
Verified postcode
Sarasota, United States
Piedmont Hospital - Piedmont Transplant Institute
Verified postcode
Atlanta, United States
Tandem Clinical Research GI
Verified postcode
Marrero, United States

Common questions

What is Primary Sclerosing Cholangitis (PSC)?

PSC is a rare liver disease where the bile ducts (small tubes in your liver) become damaged, leading to bile build-up and liver damage.

What is elafibranor?

Elafibranor is the study medicine being tested to see if it can help people with PSC. It's a new drug not yet widely available.

What is a placebo?

A placebo is a 'dummy' pill that looks just like the study medicine but doesn't contain any active drug. It helps us compare the effects of the actual medicine.

Will I know if I'm getting the active drug or the placebo?

No, during the main part of the study, neither you nor your doctor will know if you are receiving elafibranor or the placebo. This is called 'blinding' and helps ensure fair results.

How long will I be in the study?

The total length of your time in the study will be discussed by the research team. It includes a period of taking the medicine and follow-up checks.