Study on the Safety and Effectiveness of Elafibranor for Adults with Primary Sclerosing Cholangitis

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What is this study about?

This clinical trial is focused on studying a liver disease called Primary Sclerosing Cholangitis (PSC). PSC is a condition that affects the bile ducts, which are tubes that carry bile from the liver to the gallbladder and small intestine. The study is testing a treatment called elafibranor, which is taken as a film-coated tablet. Elafibranor works by activating certain proteins in the body that may help improve liver function. The study will compare the effects of elafibranor at two different doses, 80 mg and 120 mg, to a placebo, which is a tablet that does not contain any active medication.

The purpose of the study is to evaluate the safety and effectiveness of elafibranor in adults with PSC. Participants will take the medication daily for a period of 12 weeks during the initial phase of the study. After this period, there is an option for participants to continue in a long-term extension phase, where they can receive elafibranor for a longer duration to further assess its safety and tolerability. Throughout the study, participants will have regular check-ups to monitor their health and any changes in their condition.

This study aims to provide valuable information about the potential benefits and risks of using elafibranor for treating PSC. By participating, individuals with PSC can contribute to research that may lead to better treatment options for this challenging liver disease. The study is conducted under strict guidelines to ensure the safety and well-being of all participants.

1 joining the study

Upon joining the study, participants are randomly assigned to receive either elafibranor or a placebo. This process is double-blind, meaning neither the participant nor the study team knows which treatment is being administered.

The study involves taking a film-coated tablet orally. The dosage of elafibranor is either 80 mg or 120 mg, taken daily.

2 double-blind period (DBP)

During the first 12 weeks, the focus is on assessing the safety and tolerability of the treatment. Participants will continue taking their assigned tablet daily.

Regular monitoring includes physical examinations, vital signs, and laboratory tests to check liver and kidney function, among other health indicators.

3 evaluation at week 12

At the end of the 12-week period, participants undergo a comprehensive evaluation to assess changes in their condition, particularly focusing on liver function tests.

The study measures the relative and absolute changes in specific liver enzymes and other markers to determine the treatment’s impact.

4 open-label extension period (OLE)

Participants who continue into the open-label extension will receive elafibranor up to 120 mg daily. This phase is designed to evaluate the long-term safety and effects of the treatment.

The open-label phase allows all participants to know they are receiving the active medication, not a placebo.

5 long-term monitoring

Throughout the open-label extension, participants will be monitored regularly, with evaluations at weeks 52 and 96 to assess ongoing safety and liver function.

The study aims to observe any long-term changes in liver enzyme levels and overall health status.

Who Can Join the Study?

Participants must be male or female and between 18 to 75 years old at the time of signing the consent form.
Participants must have a diagnosis of Primary Sclerosing Cholangitis (PSC), which is a liver disease. This should be confirmed by:
- Historical evidence of an elevated ALP (a liver enzyme) above normal levels for at least 6 months before the first screening visit.
- A cholangiogram (a type of imaging test) showing features compatible with PSC.
During screening, participants must have an ALP level that is at least 1.5 times the upper limit of normal (ULN), with specific conditions on the variability of these levels.
Total bilirubin levels must be no more than 2 times the ULN at the first screening visit. Bilirubin is a substance made by the liver.
If taking UDCA (a medication for liver disease), participants should have:
- A total daily dose of no more than 23 mg per kg of body weight per day.
- Been on a stable treatment for at least 6 months before screening and expected to continue this dose during the 12-week study period.
- Stopped taking UDCA at least 3 months before screening if it was recently discontinued.
For participants with Inflammatory Bowel Disease (IBD):
- Those with Crohn’s disease must be in remission, meaning the disease is not active, based on the doctor’s assessment and on stable treatment before and during screening.
- Those with ulcerative colitis must be in remission or have low disease activity, as judged by the doctor, and on stable treatment before and during screening.
- Current treatment for IBD is allowed if the participant has been well-controlled for at least 3 months before screening and is expected to stay on a stable dose of IBD medications.
- A colonoscopy (a test to look inside the colon) must have been done within two years before screening, showing no signs of abnormal cell growth or cancer.
Medications for managing itching (pruritus) must be on a stable dose for at least 3 months before screening.
Participants must use contraception according to local regulations for clinical study participants.
Participants must be able to give signed informed consent, agreeing to follow the study’s requirements and restrictions.

Who Cannot Join the Study?

Patients who have a different liver condition other than Primary Sclerosing Cholangitis cannot participate. This is a specific liver disease that affects the bile ducts.
Patients who are not adults are excluded. This means only individuals who are 18 years or older can participate.
Patients who are pregnant or breastfeeding cannot take part in the study.
Patients who have participated in another clinical trial recently may not be eligible.
Patients with certain medical conditions that could interfere with the study results may be excluded.
Patients who are unable to follow the study procedures or take the study medication as required will not be able to participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
IRCCS Humanitas Research Hospital	Rozzano	Italy
Universitaetsklinikum Heidelberg AöR	Heidelberg	Germany
Unidade Local De Saude De Lisboa Ocidental E.P.E.	Carnaxide	Portugal
Unidade Local De Saúde De Santa Maria, E.P.E.	Lisbon	Portugal

Other Sites

Site Name	City	Country
Casa Sollievo Della Sofferenza	San Giovanni Rotondo	Italy
Azienda Ospedaliero Universitaria Di Modena	Modena	Italy
Hospital General Universitario Gregorio Maranon	Madrid	Spain
Hospital Universitario Puerta De Hierro De Majadahonda	Majadahonda	Spain
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone	Palermo	Italy
Hospital Universitario Rio Hortega	Valladolid	Spain
Complexo Hospitalario Universitario De Pontevedra	Pontevedra	Spain
Unidade Local De Saude Do Alto Ave E.P.E.	Guimaraes	Portugal
Azienda Ospedaliera di Padova	Padua	Italy
Charite Universitaetsmedizin Berlin KöR	Berlin	Germany
Ghdqqf Upilsonlid Folybkgig	Frankfurt	Germany
Flkfdrbkq Pdnc Ll Ictgkglmmraio Bowesqkkr Dyg Hhvcqjwp Uihgtrafvizry Lj Prk	Madrid	Spain
Hlmmxtuf Vceg dzhkvase	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Germany	Not recruiting	24.04.2023
Italy	Not recruiting	24.04.2023
Portugal	Not recruiting	24.04.2023
Spain	Not recruiting	24.04.2023

Trial locations

Investigated drugs:

Elafibranor

Elafibranor is a medication being studied for its potential to treat adults with Primary Sclerosing Cholangitis (PSC). In this clinical trial, researchers are evaluating how safe and tolerable it is for patients to take elafibranor daily. The study includes a double-blind period where neither the participants nor the researchers know who is receiving the medication or a placebo. After this period, there is an open-label extension where all participants receive elafibranor to further assess its long-term safety and effects.

Primary Sclerosing Cholangitis – This is a chronic liver disease characterized by inflammation and scarring of the bile ducts inside and outside the liver. Over time, the scarring can cause the bile ducts to become blocked, leading to a buildup of bile in the liver. This can result in liver damage and eventually lead to liver failure. The disease progresses slowly, and symptoms may not appear until significant liver damage has occurred. Common symptoms include fatigue, itching, and jaundice. The exact cause of primary sclerosing cholangitis is unknown, but it is often associated with inflammatory bowel diseases like ulcerative colitis.

Trial ID:

2024-511370-72-00

Protocol code:

CLIN-60190-453

NCT ID:

NCT05627362

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on the Safety and Effectiveness of Elafibranor for Adults with Primary Sclerosing Cholangitis

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