A Study to Assess Safety and Effectiveness of Elafibranor in Adult Participants With Primary Scle… (NCT05627362) | Clinical Trial Compass
Active — Not RecruitingPhase 2
A Study to Assess Safety and Effectiveness of Elafibranor in Adult Participants With Primary Sclerosing Cholangitis.
United States, Canada, Germany68 participantsStarted 2022-12-29
Plain-language summary
This study will evaluate the effects of elafibranor (the study drug) in participants with Primary Sclerosing Cholangitis (PSC) during the double-blind period, an initial 96 week open-label extension (OLE) period, and an optional open-label extension long-term (OLE-LT) period beyond OLE Week 96. PSC is a rare disease of the liver that leads to injury and destruction of bile ducts. Damage to bile ducts leads to buildup of bile in the liver, which then causes further damage, and leads to disease progression. This study will compare elafibranor to a placebo, a dummy treatment. The main objective of the trial will be to study the safety and side effects of the study drug, including long-term safety during the open-label extension periods. The trial will also study the study drug's effects on blood tests and other tests related to PSC disease activity.
Who can participate
Age range
18 Years – 75 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria :
The following inclusion criteria apply to entry into the double-blind period and initial open-label extension (OLE) period up to OLE Week 96, unless otherwise specified.
* Participants with a diagnosis of Primary sclerosing cholangitis (PSC) as demonstrated by the presence of the following, and in the absence of apparent causes of secondary sclerosing cholangitis: i) Historical evidence of an elevated Alkaline phosphatase (ALP) \> Upper Limit Normal (ULN) since at least 6 months prior to SV1. ii) Cholangiogram (e.g. magnetic resonance cholangiopancreatography (MRCP), endoscopic retrograde cholangiopancreatography (ERCP), percutaneous transhepatic cholangiography (PTC) with features compatible with large duct PSC.
* ALP ≥1.5x ULN during screening (with variability ≤30% based on two values).
* Total bilirubin ≤2.0x ULN at Screening Visit 1(SV1)
* Participants taking ursodeoxycholic acid (UDCA) at a total daily dose ≤23 mg/kg/day, with a minimum of 6 months of stable treatment prior to screening period and expected to remain on stable dose through the 12-week DBP. Minimum of 3 months off treatment prior to screening period if UDCA was recently discontinued.
* For participants with Inflammatory bowel disease (IBD): i) Participants with Crohn's disease must be in remission based on the investigator's clinical assessment and should be on stable treatment prior to randomisation and during screening. ii) Participants with ulcerative colitis must be in remission…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Treatment Related TEAEs, Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs)
Timeframe: Double Blind Period: Baseline up to week 12, Initial Open Label Extension (OLE) Period: Baseline up to week 100, Open-Label Extension Long-Term (OLE-LT) Period Beyond OLE Week 96: OLE-LT baseline up to OLE-LT Week 260
2
Percentage of Participants With Clinically Significant Changes in Physical Examination Findings
Timeframe: Double Blind Period: Baseline up to week 12, Initial OLE Period: Baseline up to week 100
3
Percentage of Participants With Clinically Significant changes in Laboratory Parameters (blood chemistry, hematology and coagulation)
Timeframe: Double Blind Period: Baseline up to week 12, Initial OLE Period: Baseline up to week 100
4
Percentage of Participants With Clinically Significant Changes in Vital Signs
Timeframe: Double Blind Period: Baseline up to week 12, Initial OLE Period: Baseline up to week 100
5
Percentage of Participants With Clinically Significant Changes in Electrocardiogram (ECG) Readings
Timeframe: Double Blind Period: Baseline up to week 12, Initial OLE Period: Baseline up to week 100