Clinical trial NCT06447168: A Study Observing Everyday Effectiveness and Safety of the Drug Elafibranor in Participants With Primary Biliary Cholangitis Who Are Receiving Ongoing Treatment
Official title: Prospective Non-interventional, Phase IV Multicentre Study to Assess the Effectiveness, Safety and Tolerability of Elafibranor 80 mg/Day in Participants With Primary Biliary Cholangitis Receiving Treatment in a Real-world Setting.
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Primary Biliary Cholangitis
Lead sponsor: Ipsen
Sponsor class: INDUSTRY
First posted: 2024-06-06
Enrollment: 424
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants with PBC for whom the treating physician has decided to start treatment with elafibranor 80 mg/day, as recommended in the approved indication.

Brief summary: This study will collect information from participants with Primary Biliary Cholangitis (PBC) as they use the drug elafibranor in real world setting.

PBC is a progressive rare liver disease in which tubes in the liver called bile ducts are damaged.

The liver damage in PBC may lead to scarring (cirrhosis). PBC may also be associated with multiple symptoms. Many participants with PBC may require liver transplant or may die if the disease progresses and a liver transplant is not done.

In this study the main aim is to observe the effectiveness, safety and tolerability of elafibranor in participants with PBC who are receiving treatment in real world setting. The total study duration for each participants will be 24 months.


Eligibility criteria: Inclusion Criteria:

* Participant has provided written informed consent and agrees to comply with the study protocol.
* Participant with PBC diagnosis.
* Participant naïve to elafibranor, for whom the treating physician has decided to start treatment with elafibranor.
* If a participant has a caregiver who agrees to complete the caregiver questionnaires, an informed consent should be collected from the caregiver before any data is collected.

Exclusion Criteria:

* Participant who started elafibranor treatment before baseline visit.
* Participant is currently participating in, plans to participate in or has participated in an investigational drug study or medical device study containing active substance within 30 days or five half-lives of the drug/active substance, whichever is longer, prior to baseline visit.
* Participant with known hypersensitivity to the product or to any of its excipients.
* Participant with mental instability or incompetence, such that the validity of informed consent or ability to be compliant with the study is uncertain.

Primary outcomes:
measure: Percentage of participants with response to treatment
timeFrame: At month 6
Secondary outcomes:
measure: Percentage of participants with normalization of ALP levels
timeFrame: At months 3, 6, 12, 18 and 24
measure: Percentage of participants with response to treatment
timeFrame: At months 3, 12, 18 and 24
measure: Change from baseline in liver function parameters: Serum levels of alanine aminotransferase (ALT)
timeFrame: At months 3, 6, 12, 18 and 24.
measure: Change from baseline in liver function parameters: Serum levels of Aspartate aminotransferase (AST)
timeFrame: At months 3, 6, 12, 18 and 24.
measure: Change from baseline in liver function parameters: Serum levels of Gamma-glutamyl transferase (GGT)
timeFrame: At months 3, 6, 12, 18 and 24.
measure: Change from baseline in liver function parameters: Serum levels of TB
timeFrame: At months 3, 6, 12, 18 and 24.
measure: Change from baseline in liver function parameters: Conjugated (direct) bilirubin
timeFrame: At months 3, 6, 12, 18 and 24.
measure: Change from baseline in liver function parameters: Serum levels of creatinine
timeFrame: At months 3, 6, 12, 18 and 24.
measure: Change from baseline in liver function parameters: Serum levels of albumin
timeFrame: At months 3, 6, 12, 18 and 24.
measure: Change from baseline in pruritus based on PBC Itch score
timeFrame: Monthly during 24 months.
measure: Change from baseline in fatigue based on functional assessment of chronic illness therapy-fatigue (FACIT-Fatigue) scale
timeFrame: At months 3, 6, 12, 18 and 24.
measure: Change from baseline in sleep based on Pittsburgh sleep quality index (PSQI)
timeFrame: At months 3, 6, 12, 18 and 24.
measure: Change from baseline in Quality Of Life (QoL) based on PBC-40 questionnaire
timeFrame: At months 3, 6, 12, 18 and 24.
measure: Change from baseline in QoL based on 5-Dimensional Itch scale (5-D Itch, also known as 5-D pruritus scale)
timeFrame: At months 3, 6, 12, 18 and 24
measure: Change from baseline in liver stiffness
timeFrame: At months 12 and 24.
measure: Percentage of participants experiencing Adverse Events (AEs), Adverse Events of Special Interests (AESIs) and special situations (SS).
timeFrame: From baseline to up to 24 months.
measure: Participant's satisfaction on treatment
timeFrame: At months 3, 6, 12, 18 and 24.
measure: Participant's adherence to treatment
timeFrame: Every month during 24 months.
measure: Percentage of participants with clinically significant changes in laboratory parameters
timeFrame: From baseline to up to 24 months.
measure: Percentage of participants with clinically significant changes in physical examination
timeFrame: From baseline to up to 24 months.
measure: Percentage of participants developing clinically significant changes in vital signs
timeFrame: From baseline to up to 24 months.
Other outcomes:
Locations:
city: Coronado
country: United States
facility: Southern California Research Center
state: California
city: Los Angeles
country: United States
facility: Cedars-Sinai Medical Center
state: California
city: Englewood
country: United States
facility: South Denver Gastroenterology,P.C.
state: Colorado
city: Miami
country: United States
facility: Schiff Center for Liver Diseases - University of Miami
state: Florida
city: Boston
country: United States
facility: Beth Israel Deaconess Medical Center, Liver Research Center
state: Massachusetts
city: Cherry Hill
country: United States
facility: Virtua Center for Liver Disease - Cherry Hill
state: New Jersey
city: Manhasset
country: United States
facility: Northwell Health Inc, Center for Liver Disease and Transplantation
state: New York
city: Cleveland
country: United States
facility: Cleveland Clinic Foundation
state: Ohio
city: Dallas
country: United States
facility: Liver Center of Texas
state: Texas
city: Houston
country: United States
facility: Baylor College of Medicine - Advanced Liver Therapies
state: Texas
city: Katy
country: United States
facility: Velocity Clinical Research
state: Texas
city: Richmond
country: United States
facility: Bon Secours Richmond Community Hospital LLC. d/b/a Bon Secours Liver Institute of Richmond
state: Virginia
city: Richmond
country: United States
facility: Virginia Commonwealth University Medical Center - West Hospital
state: Virginia
city: Seattle
country: United States
facility: Gastro health & Nutrition
state: Washington
city: Graz
country: Austria
facility: Medizinische Universitaetsklinik Graz
city: Innsbruck
country: Austria
facility: Medical University Innsbruck
city: Calgary
country: Canada
facility: University of Calgary
city: Berlin
country: Germany
facility: Charite Universitatsmedizin Berlin
city: Berlin
country: Germany
facility: DRK Kliniken Berlin Mitte
city: Frankfurt
country: Germany
facility: Klinikum der Johann Wolfgang Goethe-Universitaet
city: Halle
country: Germany
facility: Studiengesellschaft BSF
city: Herne
country: Germany
facility: Gastroenterologsiche Studiengesellschaft Herne
city: Homburg
country: Germany
facility: Universitaet des Saarlandes
city: Leipzig
country: Germany
facility: Universitaetsklinikum Leipzig
city: Trier
country: Germany
facility: Hospital of the Merciful Brothers Trier
city: Athens
country: Greece
facility: General Hospital of Athens Laiko
city: Heraklion
country: Greece
facility: University Hospital of Heraklion
city: Patras
country: Greece
facility: University General Hospital of Patras
city: Aberdeen
country: United Kingdom
facility: Aberdeen Royal Infirmary NHS Grampian Grampian Health Board
city: Birmingham
country: United Kingdom
facility: Queen Elizabeth Hospital Birmingham - University Hospitals Birmingham NHS Foundation Trust
city: Bradford
country: United Kingdom
facility: Bradford Royal Infirmary - Bradford Teaching Hospitals NHS Foundation
city: Hull
country: United Kingdom
facility: Hull Royal Infirmary - Hull University Teaching Hospitals NHS Trust
city: London
country: United Kingdom
facility: King's College Hospital NHS Foundation Trust
city: Newcastle
country: United Kingdom
facility: The Newcastle upon Tyne Hospitals NHS Foundation Trust - Freeman Hospital
city: Nottingham
country: United Kingdom
facility: Queen's Medical Centre - Nottingham University Hospitals NHS Trust
city: Oxford
country: United Kingdom
facility: John Radcliffe Hospital - Oxford University Hospitals NHS Foundation Trust
Study officials:
affiliation: Ipsen
name: Ipsen Medical Director
role: STUDY_DIRECTOR
References: