What side effects are associated with this type of intervention?

Common treatments for Primary Sclerosing Cholangitis (PSC) include glucocorticoids, thiopurines, ursodeoxycholic acid (UDCA), and vancomycin. Glucocorticoids can cause side effects such as weight gain, osteoporosis, and increased risk of infections. Thiopurines may lead to bone marrow suppression, liver toxicity, and increased risk of infections. UDCA is generally well-tolerated but can cause diarrhea and, at higher doses, may be detrimental. Vancomycin's side effects include nephrotoxicity and ototoxicity. Elafibranor, a PPAR alpha/delta agonist, is being studied for its safety and effectiveness, with potential side effects including gastrointestinal disturbances and liver enzyme abnormalities.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for Primary Sclerosing Cholangitis (PSC). The management of PSC primarily involves supportive and palliative care, with no therapies proven to alter the disease's natural history. Ursodeoxycholic acid (UDCA) is commonly used to improve liver enzyme levels, but its efficacy in changing disease progression is not established. Elafibranor, a PPAR alpha/delta agonist, is currently being studied for its potential benefits in PSC, but it has not yet received FDA approval for this indication.

What other types of research exist?

Promising, novel alternatives to Elafibranor for Primary Sclerosing Cholangitis patients may include other PPAR agonists, anti-fibrotic agents, and drugs targeting bile acid metabolism. Specific alternatives are not detailed in the provided context, so discussing with a healthcare provider for the latest clinical trial options and emerging treatments is recommended.

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PPAR agonist

Elafibranor for Primary Sclerosing Cholangitis (ELMWOOD Trial)

Verified Trial

Phase 2

Waitlist Available

Research Sponsored by Ipsen

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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.

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up pre-dose, 0.5 hour (h), 1h, between 1.5 hours and 2h, 4h, and 6h after dosing at week 4

Summary

This trial is testing elafibranor, a medication, in people with Primary Sclerosing Cholangitis (PSC), a rare liver disease. PSC causes bile ducts to get damaged, leading to more liver problems. The study will evaluate if elafibranor is safe and effective in reducing liver inflammation and improving bile flow.

Who is the study for?

Adults with Primary Sclerosing Cholangitis (PSC) can join this trial. They must have stable Inflammatory Bowel Disease if present, no recent severe liver issues or cancer, and be on a steady dose of certain medications like ursodeoxycholic acid. Participants need to use approved contraception methods and not be pregnant or breastfeeding.

What is being tested?

The trial is testing Elafibranor at two different doses (80 mg and 120 mg) against placebos matched for each dose. The goal is to see how safe Elafibranor is and how well it works in treating PSC by comparing its effects on liver function tests and other disease-related measures.

What are the potential side effects?

While the specific side effects of Elafibranor are not listed here, common side effects may include digestive issues, fatigue, itching (pruritus), abnormal blood tests reflecting liver function changes, muscle pain or weakness.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have been diagnosed with PSC and have had high ALP levels for over 6 months.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ pre-dose, 0.5 hour (h), 1h, between 1.5h and 2h, 4h, and 6h after dosing at week 4

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~pre-dose, 0.5 hour (h), 1h, between 1.5h and 2h, 4h, and 6h after dosing at week 4

This trial's timeline: 3 weeks for screening, Varies for treatment, and pre-dose, 0.5 hour (h), 1h, between 1.5h and 2h, 4h, and 6h after dosing at week 4 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Percentage of Participants With Clinically Significant Changes in Electrocardiogram (ECG) Readings

Percentage of Participants With Clinically Significant Changes in Physical Examination Findings

Percentage of Participants With Clinically Significant Changes in Vital Signs

+2 more

Secondary study objectives

PK of Elafibranor and its Metabolite GFT1007: Time to Maximum Observed Drug Concentration (Tmax)

PK of Elafibranor and its Metabolite GFT1007: Total Body Clearance (Cl/F)

PK of Elafibranor and its Metabolite GFT1007: Volume of distribution (Vz)

+1 more

Trial Design

4Treatment groups

Experimental Treatment

Placebo Group

Group I: Open-Label Extension Period: Elafibranor 120 mgExperimental Treatment1 Intervention

Participant will receive one tablet per day (elafibranor 120 mg) over the 96 weeks in Open-Label extension period.

Group II: Double-Blind Period: Elafibranor 80 mgExperimental Treatment2 Interventions

Participant will receive two tablets per day (one tablet of elafibranor 80 mg + 1 tablet of placebo matching the 120 mg sized tablet) over the 12 weeks in Double-blind period.

Group III: Double-Blind Period: Elafibranor 120 mgExperimental Treatment2 Interventions

Participant will receive 2 tablets per day (one tablet of elafibranor 120 mg + 1 tablet of placebo matching the 80 mg sized tablet) over the 12 weeks in Double-blind period.

Group IV: Double-Blind Period: PlaceboPlacebo Group1 Intervention

Participant will receive 2 placebo tablets per day (one matching the 80 mg sized tablet + one matching the 120 mg sized tablet) over the 12 weeks in Double-blind period.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Elafibranor 80 mg

2017

Completed Phase 2

~50

Elafibranor 120 mg

2017

Completed Phase 2

~50

0 / 1Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Primary Sclerosing Cholangitis (PSC) treatments often target the regulation of bile acid metabolism and inflammation. Elafibranor, a PPAR alpha/delta agonist, works by activating peroxisome proliferator-activated receptors (PPARs), which play a crucial role in lipid metabolism, inflammation reduction, and bile acid detoxification. Similar treatments, such as fibrates (e.g., fenofibrate and bezafibrate), also activate PPARs, leading to improved liver biochemistries and reduced bile acid toxicity. These mechanisms are significant for PSC patients as they help manage disease progression by reducing bile duct inflammation and damage, ultimately aiming to preserve liver function and delay the onset of severe complications.

Peroxisome proliferator-activated receptor α activates human multidrug resistance transporter 3/ATP-binding cassette protein subfamily B4 transcription and increases rat biliary phosphatidylcholine secretion.Effects of three different fibrates on intrahepatic cholestasis experimentally induced in rats.