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

Phase 2

Active, not recruiting

• Conditions: Primary Sclerosing Cholangitis
• Interventions: Drug: Elafibranor 120 mg; Drug: Elafibranor 80 mg; Drug: Placebo Matched to Elafibranor 120 mg; Drug: Placebo Matched to Elafibranor 80 mg

• Registration Number: NCT05627362
• Lead Sponsor: Ipsen

Brief Summary

This study will evaluate the effects of elafibranor (the study drug) in participants with Primary Sclerosing Cholangitis (PSC). 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. The trial will also study the study drug's effects on blood tests and other tests related to PSC disease activity.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-11-22
• Study First Submitted QC: 2022-11-23
• Study First Posted: 2022-11-25
• Study Start: 2022-12-29
• Status Verified: 2025-06
• Last Update Submitted: 2025-06-18
• Last Update Posted: 2025-06-19
• Primary Completion: 2026-08-28
• Study Completion: 2026-08-28

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 68

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Double-Blind Period: Elafibranor 80 mg	Placebo Matched to Elafibranor 120 mg	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.
Double-Blind Period: Elafibranor 120 mg	Placebo Matched to Elafibranor 80 mg	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.
Double-Blind Period: Placebo	Placebo Matched to Elafibranor 80 mg	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.
Open-Label Extension Period: Elafibranor 120 mg	Elafibranor 120 mg	Participant will receive one tablet per day (elafibranor 120 mg) over the 96 weeks in Open-Label extension period.
Double-Blind Period: Elafibranor 120 mg	Elafibranor 120 mg	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.
Double-Blind Period: Elafibranor 80 mg	Elafibranor 80 mg	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.

Primary Outcome Measures

Name	Time	Method
Percentage of Participants With Treatment Emergent Adverse Event (TEAEs), Treatment Related TEAEs, Serious Adverse (SAEs) and Adverse Events of Special Interest (AESIs)	Double Blind Period: Baseline up to week 12, Open Label Extension (OLE) Period: Baseline up to week 100	An Adverse event (AE) is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. AESIs are AEs that may not be serious but are of special importance to a particular drug or class of drugs.
Percentage of Participants With Clinically Significant Changes in Physical Examination Findings	Double Blind Period: Baseline up to week 12, OLE Period: Baseline up to week 100	Percentage of participants with clinically significant changes in physical examination findings will be reported. The clinical significance will be decided by the investigator.
Percentage of Participants With Clinically Significant changes in Laboratory Parameters (blood chemistry, hematology and coagulation)	Double Blind Period: Baseline up to week 12, OLE Period: Baseline up to week 100	Percentage of participants with clinically significant change in laboratory parameters (blood chemistry, hematology and coagulation) will be reported. The clinical significance will be decided by the investigator.
Percentage of Participants With Clinically Significant Changes in Vital Signs	Double Blind Period: Baseline up to week 12, OLE Period: Baseline up to week 100	Percentage of participants with clinically significant changes in Vital Signs will be reported. The clinical significance will be decided by the investigator.
Percentage of Participants With Clinically Significant Changes in Electrocardiogram (ECG) Readings	Double Blind Period: Baseline up to week 12, OLE Period: Baseline up to week 100	Percentage of participants with clinically significant changes in ECG readings will be reported. The clinical significance will be decided by the investigator.

Secondary Outcome Measures

Name	Time	Method
Relative Change From Baseline in Alkaline Phosphate Levels (ALP)	Double Blind Period: Baseline, Week 12, OLE Period: Baseline, Week 52, Week 96
Percentage of Participants With ≥40% Decrease from Baseline in ALP Levels	Double Blind Period: Baseline, Week 12, OLE Period: Baseline, Week 52, Week 96
Absolute Change from Baseline in ALP	Double Blind Period: Baseline, Week 12, OLE Period: Baseline, Week 52, Week 96
Percentage of Participants With ALP: <1.3x Upper Limit of Normal (ULN) and <1.5x ULN	Double Blind Period: Week 12
Percentage of Participants who Normalised ALP	Double Blind Period: Week 12
Change From Baseline in Alanine Transaminase (ALT),Aspartate Transaminase (AST), Gamma-glutamyl transferase (GGT), 5' Nucleotidase and Fractionated ALP Levels at Week 12	Baseline, Week 12
Change From Baseline in Total bilirubin, Conjugated bilirubin Levels at Week 12	Baseline, Week 12
Change From Baseline in Albumin Levels at Week 12	Baseline, Week 12
Change from Baseline in Enhanced Liver Fibrosis (ELF) Test Score	Double Blind Period: Baseline, Week 12
Change From Baseline in Liver Stiffness Measurement (LSM) Values Assessed by FibroScan® at Week 12	Double Blind Period: Baseline, Week 12
Change From Baseline in Other Non-invasive Hepatic Fibrosis Serum Markers as Measured by PAI-1, TGF-β, Marker of type V Collagen Formation (Pro-C5), and Marker of Type III Collagen Formation (Pro-C3)	Double Blind Period: Baseline, Week 12
Change From Baseline in Fibrosis-4 (FIB-4) and AST to Platelet Ratio Index (APRI)	Double Blind Period: Baseline, Week 12
Change From Baseline in Cytokeratin-18 (CK-18) (M65 and M30) Levels	Double Blind Period: Baseline, Week 12
Pharmacokinetics (PK) of Elafibranor and its Metabolite GFT1007: Area Under the Concentration-time Curve Over the Dosing Interval from Time 0 to 24 hours(AUC0-24)	Pre-dose, 0.5 hour (h), 1h, between 1.5 hours and 2h, 4h, and 6h after dosing at Week 4	AUC 0-24 will be recorded from the PK blood samples collected.
PK of Elafibranor and its Metabolite GFT1007: Maximum (peak) Observed Plasma Drug Concentration (Cmax)	Pre-dose, 0.5 hour (h), 1h, between 1.5h and 2h, 4h, and 6h after dosing at Week 4
PK of Elafibranor and its Metabolite GFT1007: Time to Maximum Observed Drug Concentration (Tmax)	Pre-dose, 0.5 hour (h), 1h, between 1.5h and 2h, 4h, and 6h after dosing at Week 4	Tmax will be recorded from the PK blood samples collected.
PK of Elafibranor and its Metabolite GFT1007: Total Body Clearance (Cl/F)	Double Blind Period: Baseline up to Week 12	Cl/F will be recorded from the PK blood samples collected.
PK of Elafibranor and its Metabolite GFT1007: Volume of distribution (Vz)	Double Blind Period: Baseline up to Week 12	Vz will be recorded from the PK blood samples collected.

Trial Locations

Locations (62)

Om Research LLC; 🇺🇸 Lancaster, California, United States

Cedars-Sinai Medical Center; 🇺🇸 Los Angeles, California, United States

University of California, Davis; 🇺🇸 Sacramento, California, United States

Sutter Health Van Ness Campus Medical Office Building; 🇺🇸 San Francisco, California, United States

Peak Gastroenterology Associates; 🇺🇸 Colorado Springs, Colorado, United States

South Denver Gastroenterology,P.C.; 🇺🇸 Englewood, Colorado, United States

Rocky Mountain Gastroenterology (RMG); 🇺🇸 Littleton, Colorado, United States

Yale University School Of Medicine - Yale Center For Clinical Investigation; 🇺🇸 New Haven, Connecticut, United States

Schiff Center for Liver Diseases - University of Miami; 🇺🇸 Miami, Florida, United States

Covenant Research; 🇺🇸 Sarasota, Florida, United States

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