A Study Observing Everyday Effectiveness and Safety of the Drug Elafibranor in Participants With Primary Biliary Cholangitis Who Are Receiving Ongoing Treatment

Primary Biliary Cholangitis Clinical Trial

— ELFINITY  

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.

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06447168
• Other study ID #: CLIN-60190-461
• Status: Not yet recruiting
• Start date: July 15, 2024
• Est. completion date: July 15, 2029

Study information

• Verified date: June 2024
• Source: Ipsen
• Contact: Ipsen Clinical Study Enquiries
• Phone: See e mail
• Email: clinical.trials[@]ipsen.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 424
• Est. completion date: July 15, 2029
• Est. primary completion date: July 15, 2029
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Cholangitis
• Liver Cirrhosis, Biliary
• Primary Biliary Cholangitis

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Ipsen

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percentage of participants with response to treatment	Defined as alkaline phosphatase (ALP) <1.67 x upper limit of normal (ULN) and total bilirubin (TB) =ULN and ALP decrease =15% from baseline.	At month 6
Secondary	Percentage of participants with normalization of ALP levels		At months 3, 6, 12, 18 and 24
Secondary	Percentage of participants with response to treatment	Defined as ALP<1.67 x ULN and TB=ULN and ALP decrease =15% from baseline.	At months 3, 12, 18 and 24
Secondary	Change from baseline in liver function parameters: Serum levels of alanine aminotransferase (ALT)		At months 3, 6, 12, 18 and 24.
Secondary	Change from baseline in liver function parameters: Serum levels of Aspartate aminotransferase (AST)		At months 3, 6, 12, 18 and 24.
Secondary	Change from baseline in liver function parameters: Serum levels of Gamma-glutamyl transferase (GGT)		At months 3, 6, 12, 18 and 24.
Secondary	Change from baseline in liver function parameters: Serum levels of TB		At months 3, 6, 12, 18 and 24.
Secondary	Change from baseline in liver function parameters: Conjugated (direct) bilirubin		At months 3, 6, 12, 18 and 24.
Secondary	Change from baseline in liver function parameters: Serum levels of creatinine		At months 3, 6, 12, 18 and 24.
Secondary	Change from baseline in liver function parameters: Serum levels of albumin		At months 3, 6, 12, 18 and 24.
Secondary	Change from baseline in pruritus based on PBC Itch score	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).	Monthly during 24 months.
Secondary	Change from baseline in fatigue based on functional assessment of chronic illness therapy-fatigue (FACIT-Fatigue) scale	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'.	At months 3, 6, 12, 18 and 24.
Secondary	Change from baseline in sleep based on Pittsburgh sleep quality index (PSQI)	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.	At months 3, 6, 12, 18 and 24.
Secondary	Change from baseline in Quality Of Life (QoL) based on PBC-40 questionnaire	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.	At months 3, 6, 12, 18 and 24.
Secondary	Change from baseline in QoL based on 5-Dimensional Itch scale (5-D Itch, also known as 5-D pruritus scale)	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.	At months 3, 6, 12, 18 and 24
Secondary	Change from baseline in liver stiffness	Measured by transient elastography (FibroScan®) and enhanced liver fibrosis (ELF) test	At months 12 and 24.
Secondary	Percentage of participants experiencing Adverse Events (AEs), Adverse Events of Special Interests (AESIs) and special situations (SS).	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.	From baseline to up to 24 months.
Secondary	Participant's satisfaction on treatment	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).	At months 3, 6, 12, 18 and 24.
Secondary	Participant's adherence to treatment	Adherence to the treatment will be measured based on the participant report of missed doses every month.	Every month during 24 months.
Secondary	Percentage of participants with clinically significant changes in laboratory parameters	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.	From baseline to up to 24 months.
Secondary	Percentage of participants with clinically significant changes in physical examination	Clinically significant changes in physical examination will be reported. The clinical significance will be graded by the investigator.	From baseline to up to 24 months.
Secondary	Percentage of participants developing clinically significant changes in vital signs	Clinically significant changes in vital signs will be reported. The clinical significance will be graded by the investigator.	From baseline to up to 24 months.