A Study to Assess Safety and Effectiveness of Elafibranor in Adult Participants With Primary Sclerosing Cholangitis.
- Conditions
- Primary Sclerosing Cholangitis
- Interventions
- Drug: Placebo Matched to Elafibranor 120 mgDrug: 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
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 68
Not provided
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.
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Trial Locations
- Locations (62)
Cedars-Sinai Medical Center
🇺🇸Los Angeles, California, United States
Piedmont Hospital - Piedmont Transplant Institute
🇺🇸Atlanta, Georgia, United States
Aberdeen Royal Infirmary NHS Grampian Grampian Health Board
🇬🇧Aberdeen, United Kingdom
Penn State Milton S Hershey Medical Center
🇺🇸Hershey, Pennsylvania, United States
Schiff Center for Liver Diseases - University of Miami
🇺🇸Miami, Florida, United States
Medical University of South Carolina
🇺🇸Charleston, South Carolina, United States
Rocky Mountain Gastroenterology (RMG)
🇺🇸Littleton, Colorado, United States
Brampton Civic Hospital (BCH) - Osler Hepatitis Centre
🇨🇦Brampton, Ontario, Canada
Mercy Medical Center
🇺🇸Baltimore, Maryland, United States
Virginia Commonwealth University
🇺🇸Richmond, Virginia, United States
University Of Alberta Hospital-Zeidler Ledcor Centre
🇨🇦Edmonton, Alberta, Canada
Ospedale Casa Sollievo della Sofferenza
🇮🇹San Giovanni Rotondo, Italy
Tandem Clinical Research GI
🇺🇸Marrero, Louisiana, United States
University of Virginia Medical Center
🇺🇸Charlottesville, Virginia, United States
University Of Texas Southwestern Medical Center At Dallas
🇺🇸Dallas, Texas, United States
Centro Hospitalar de Lisboa ocidental (CHLO), Hospital Egas Moniz
🇵🇹Lisboa, Portugal
Hospital General Universitario Gregorio Maranon (HGUGM)
🇪🇸Madrid, Spain
Gastro Health Research
🇺🇸Cincinnati, Ohio, United States
Centre de Recherche du Centre Hospitalier de l'Universite de Montreal
🇨🇦Montréal, Canada
Hospital Universitario Puerta de Hierro
🇪🇸Majadahonda, Spain
Hospital De Montecelo
🇪🇸Pontevedra, Spain
Centro Hospitalar Universitario Lisboa Norte
🇵🇹Lisboa, Portugal
Intermountain Medical Center
🇺🇸Murray, Utah, United States
Hospital Universitario Miguel Servet
🇪🇸Zaragoza, Spain
Gruppo Humanitas - Humanitas Research Hospital, Istituto Clinico Humanitas
🇮🇹Rozzano, Italy
Hospital Universitario La Paz
🇪🇸Madrid, Spain
Queen Elizabeth Hospital
🇬🇧Birmingham, United Kingdom
Frimley Park Hospital - Frimley Health NHS Foundation Trust
🇬🇧Frimley, United Kingdom
Hospital Universitario Vall d'Hebron
🇪🇸Barcelona, Spain
Hospital Universitario Rio Hortega
🇪🇸Valladolid, Spain
Beth Israel Deaconess Medical Center, Liver Research Center
🇺🇸Boston, Massachusetts, United States
Liver Institute Northwest
🇺🇸Seattle, Washington, United States
American Research Corporation at The Texas Liver Institute
🇺🇸San Antonio, Texas, United States
Sutter Health Van Ness Campus Medical Office Building
🇺🇸San Francisco, California, United States
Hull Royal Infirmary - Hull University Teaching Hospitals NHS Trust
🇬🇧Hull, United Kingdom
University of Calgary
🇨🇦Calgary, Alberta, Canada
University of Nebraska Medical Center
🇺🇸Omaha, Nebraska, United States
Om Research LLC
🇺🇸Lancaster, California, United States
Azienda Ospedaliero Universitaria Modena
🇮🇹Modena, Italy
New York University Langone Health
🇺🇸New York, New York, United States
Huron Gastroenterology Associates - Center for Digestive Care
🇺🇸Ypsilanti, Michigan, United States
Southwest Gastroenterology Associates, PC (SWGA)
🇺🇸Albuquerque, New Mexico, United States
Gastro One
🇺🇸Cordova, Tennessee, United States
Bon Secours Richmond Community Hospital LLC. d/b/a Bon Secours Liver Institute of Richmond
🇺🇸Richmond, Virginia, United States
Aspen Woods Clinic
🇨🇦Calgary, Canada
Klinikum der Johann Wolfgang Goethe-Universitaet Frankfurt
🇩🇪Frankfurt, Germany
G.I Research Institute
🇨🇦Vancouver, Canada
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
🇮🇹Palermo, Italy
Universitaetsklinikum Heidelberg - Nationales Centrum fuer Tumorerkrankungen
🇩🇪Heidelberg, Germany
Azienda Ospedaliera di Padova - U.O.C. di Gastroenterologia
🇮🇹Padova, Italy
Charite Campus Virchow
🇩🇪Berlin, Germany
Centro Hospitalar do Alto Ave - Hospital Senhora da Oliveira
🇵🇹Guimarães, Portugal
Glasgow Royal Infirmary - Greater Glasgow Health Board
🇬🇧Glasgow, United Kingdom
The Royal Free Hospital - Royal Free London NHS Foundation Trust
🇬🇧London, United Kingdom
Ambrose King Centre-Royal London Hospital-Barts Health NHS Trust
🇬🇧London, United Kingdom
South Denver Gastroenterology,P.C.
🇺🇸Englewood, Colorado, United States
Yale University School Of Medicine - Yale Center For Clinical Investigation
🇺🇸New Haven, Connecticut, United States
University of California, Davis
🇺🇸Sacramento, California, United States
Peak Gastroenterology Associates
🇺🇸Colorado Springs, Colorado, United States
Covenant Research
🇺🇸Sarasota, Florida, United States
University Hospital Ulm
🇩🇪Ulm, Germany
Thomas Jefferson University
🇺🇸Philadelphia, Pennsylvania, United States