A Study Observing Everyday Effectiveness and Safety of the Drug Elafibranor in Participants With Primary Biliary Cholangitis Who Are Receiving Ongoing Treatment
- Conditions
- Primary Biliary Cholangitis
- Registration Number
- NCT06447168
- Lead Sponsor
- Ipsen
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 424
- 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.
- 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.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Percentage of participants with response to treatment At month 6 Defined as alkaline phosphatase (ALP) \<1.67 x upper limit of normal (ULN) and total bilirubin (TB) ≤ULN and ALP decrease ≥15% from baseline.
- Secondary Outcome Measures
Name Time Method Percentage of participants experiencing Adverse Events (AEs), Adverse Events of Special Interests (AESIs) and special situations (SS). From baseline to up to 24 months. An Adverse event (AE) is any untoward medical occurrence, temporally associated with the use of study intervention, whether or not 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.
Participant's satisfaction on treatment At months 3, 6, 12, 18 and 24. Measured by treatment satisfaction questionnaire for medication (TSQM). The TSQM (version 1.4) has 14 questions divided into 4 subscales: effectiveness (items 1 to 3), side effects (items 4 to 8), convenience (items 9 to 11), and global satisfaction (items 12 to 14).
Participant's adherence to treatment Every month during 24 months. Adherence to the treatment will be measured based on the participant report of missed doses every month.
Change from baseline in sleep based on Pittsburgh sleep quality index (PSQI) At months 3, 6, 12, 18 and 24. The PSQI was designed to evaluate overall sleep quality in the psychiatric disorders associated with sleep disturbances. Each of the questionnaire's 19-self-reported items belongs to one of seven subcategories: subjective sleep quality, sleep latency, sleep duration, habitual sleep efficiency, sleep disturbances, use of sleeping medication, and daytime dysfunction. Five additional questions rated by the respondent's roommate or bed partner are included for clinical purposes and are not scored.
Change from baseline in liver function parameters: Serum levels of creatinine At months 3, 6, 12, 18 and 24. Change from baseline in pruritus based on PBC Itch score Monthly during 24 months. The PBC Itch score is a simple, self-administered Patient Reported Outcome (PRO) questionnaire that measures itch intensity. It uses 7-day recall periods and asks participants to rate the intensity of their worst itch over the past 7-day period on an 11-point scale ranging from 0 (no itch) to 10 (worst itch imaginable).
Percentage of participants with normalization of ALP levels At months 3, 6, 12, 18 and 24 Change from baseline in liver function parameters: Serum levels of TB At months 3, 6, 12, 18 and 24. Percentage of participants with response to treatment At months 3, 12, 18 and 24 Defined as ALP\<1.67 x ULN and TB≤ULN and ALP decrease ≥15% from baseline.
Change from baseline in liver function parameters: Serum levels of alanine aminotransferase (ALT) At months 3, 6, 12, 18 and 24. Change from baseline in liver function parameters: Serum levels of Aspartate aminotransferase (AST) At months 3, 6, 12, 18 and 24. Change from baseline in liver function parameters: Serum levels of Gamma-glutamyl transferase (GGT) At months 3, 6, 12, 18 and 24. Change from baseline in liver function parameters: Conjugated (direct) bilirubin At months 3, 6, 12, 18 and 24. Change from baseline in liver function parameters: Serum levels of albumin At months 3, 6, 12, 18 and 24. Change from baseline in fatigue based on functional assessment of chronic illness therapy-fatigue (FACIT-Fatigue) scale At months 3, 6, 12, 18 and 24. The FACIT-Fatigue is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function. It is a subset of the longer (47-item) Functional Assessment of Cancer Therapy - Anemia (FACT-An), which includes the 27-item FACT-G and a 20-item subscale addressing additional concerns associated with the anemia of cancer and its treatment. This 20-item subscale, referred to as the anemia subscale, is comprised of 13-items that assess fatigue and its impact (the FACIT-Fatigue) and, 7 additional symptoms associated with anemia (e.g. shortness of breath, headache). participants rate their symptoms over the preceding seven days on a verbal response scale, the options range from 'not at all' / 'a little bit' / 'somewhat quite a bit' / very much'.
Change from baseline in Quality Of Life (QoL) based on PBC-40 questionnaire At months 3, 6, 12, 18 and 24. The PBC-40 is a validated, PBC-specific, health-related Quality Of Life (QoL) questionnaire with 40 questions that assesses symptoms across six domains: fatigue, emotional and social, cognitive function, general symptoms and itch. Participants respond on a verbal response scale, depending on the section options range from 'never' / 'not at all' / 'strongly disagree' to 'always' / 'very much'/ 'strongly agree'. Five items (3/3 in the itch domain and 2/10 in the social domain) also include a 'does not apply' option. A score for each domain is provided (but a total score is not calculated), with each verbal response scale correlating to a score of 1-5 per item (0-5 on items with a 'does not apply' option) with 5 being the most affected.
Change from baseline in QoL based on 5-Dimensional Itch scale (5-D Itch, also known as 5-D pruritus scale) At months 3, 6, 12, 18 and 24 The 5-D Itch scale assesses symptoms in terms of five domains: degree, duration, direction, disability and distribution. It is a 1 to 5 scale, with 5 being the most affected.
Change from baseline in liver stiffness At months 12 and 24. Measured by transient elastography (FibroScan®) and enhanced liver fibrosis (ELF) test
Percentage of participants with clinically significant changes in laboratory parameters From baseline to up to 24 months. Percentage of participants with clinically significant change in laboratory parameters (blood chemistry, hematology and coagulation) will be reported. The clinical significance will be graded by the investigator.
Percentage of participants developing clinically significant changes in vital signs From baseline to up to 24 months. Clinically significant changes in vital signs will be reported. The clinical significance will be graded by the investigator.
Percentage of participants with clinically significant changes in physical examination From baseline to up to 24 months. Clinically significant changes in physical examination will be reported. The clinical significance will be graded by the investigator.
Trial Locations
- Locations (33)
Medizinische Universitaetsklinik Graz
🇦🇹Graz, Austria
Medical University Innsbruck
🇦🇹Innsbruck, Austria
Hospital of the Merciful Brothers Trier
🇩🇪Trier, Germany
General Hospital of Athens Laiko
🇬🇷Athens, Greece
University Hospital of Heraklion
🇬🇷Heraklion, Greece
Studiengesellschaft BSF
🇩🇪Halle, Germany
Gastroenterologsiche Studiengesellschaft Herne
🇩🇪Herne, Germany
Universitaet des Saarlandes
🇩🇪Homburg, Germany
Universitaetsklinikum Leipzig
🇩🇪Leipzig, Germany
Klinikum der Johann Wolfgang Goethe-Universitaet
🇩🇪Frankfurt, Germany
Beth Israel Deaconess Medical Center, Liver Research Center
🇺🇸Boston, Massachusetts, United States
Southern California Research Center
🇺🇸Coronado, California, United States
South Denver Gastroenterology,P.C.
🇺🇸Englewood, Colorado, United States
Schiff Center for Liver Diseases - University of Miami
🇺🇸Miami, Florida, United States
Northwell Health Inc, Center for Liver Disease and Transplantation
🇺🇸Manhasset, New York, United States
Cleveland Clinic Foundation
🇺🇸Cleveland, Ohio, United States
Liver Center of Texas
🇺🇸Dallas, Texas, United States
Baylor College of Medicine - Advanced Liver Therapies
🇺🇸Houston, Texas, United States
Velocity Clinical Research
🇺🇸Katy, Texas, United States
Bon Secours Richmond Community Hospital LLC. d/b/a Bon Secours Liver Institute of Richmond
🇺🇸Richmond, Virginia, United States
Virginia Commonwealth University Medical Center - West Hospital
🇺🇸Richmond, Virginia, United States
Gastro health & Nutrition
🇺🇸Seattle, Washington, United States
University of Calgary
🇨🇦Calgary, Canada
Charite Universitatsmedizin Berlin
🇩🇪Berlin, Germany
DRK Kliniken Berlin Mitte
🇩🇪Berlin, Germany
University General Hospital of Patras
🇬🇷Patras, Greece
Aberdeen Royal Infirmary NHS Grampian Grampian Health Board
🇬🇧Aberdeen, United Kingdom
Queen Elizabeth Hospital Birmingham - University Hospitals Birmingham NHS Foundation Trust
🇬🇧Birmingham, United Kingdom
Bradford Royal Infirmary - Bradford Teaching Hospitals NHS Foundation
🇬🇧Bradford, United Kingdom
Hull Royal Infirmary - Hull University Teaching Hospitals NHS Trust
🇬🇧Hull, United Kingdom
King's College Hospital NHS Foundation Trust
🇬🇧London, United Kingdom
The Newcastle upon Tyne Hospitals NHS Foundation Trust - Freeman Hospital
🇬🇧Newcastle, United Kingdom
John Radcliffe Hospital - Oxford University Hospitals NHS Foundation Trust
🇬🇧Oxford, United Kingdom