Prospective Observational Study Assessing the Impact of Elafibranor on Fatigue, Work Productivity/Activity Impairment, and Pruritus in Patients With PBC

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Evaluation of patient-reported outcomes
• Data collection methods:
  Combined primary data collection and secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medicinal product name:
  IQIRVO
• Medicinal product name, other:
  ELAFIBRANOR
• Study drug International non-proprietary name (INN) or common name:
  ELAFIBRANOR
• Anatomical Therapeutic Chemical (ATC) code:
  (A05AX06) elafibranor

  elafibranor
• Medical condition to be studied:
  Primary biliary cholangitis

Population studied

• Short description of the study population:
  The participants of this study will be adult participants in the United States with a documented diagnosis of PBC who are newly initiating elafibranor in routine clinical practice
• Age groups:
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 65 years)

      • Adults (65 to < 75 years)
      • Adults (75 to < 85 years)
      • Adults (85 years and over)
• Special population of interest:
  Hepatic impaired
• Estimated number of subjects:
  50

Study design details

• Study design:
  This is a decentralized non-interventional, descriptive, real-world study of adult participants with primary biliary cholangitis (PBC) who are newly initiating elafibranor in routine clinical practice in the US. Data will be collected through medical record retrieval and ePRO assessments.
• Main study objective:
  To evaluate participant-reported fatigue at baseline (pre-initiation of elafibranor) and through Month 3, Month 6, and Month 12 (M3, M6, and M12) after initiation of elafibranor for treatment of PBC (reference date).
• Outcomes:
  Primary Outcome Measure:; 1.Change From Baseline in PBC-40 Fatigue Domain Score; 2.Change From Baseline in PROMIS Fatigue Short Form 7a Score; ; Secondary Outcome Measures:; Baseline Demographic and Clinical Characteristics of Participants; Change From Baseline in WPAI-SHP Absenteeism Score; Change From Baseline in WPAI-SHP Presenteeism Score; Change From Baseline in WPAI-SHP Overall Work Impairment Score; Change From Baseline in WPAI-SHP Activity Impairment Score; Change From Baseline in PBC-40 Emotional Domain Score; Change From Baseline in PBC-40 Social Domain Score; Change From Baseline in PBC-40 Cognitive Domain Score; Change From Baseline in PBC-40 General Symptoms Domain Score; Change From Baseline in PBC-40 Itch Domain Score; Change From Baseline in Worst Itch Numeric Rating Scale (WI-NRS) Score
• Data analysis plan:
  This study is descriptive in nature. Analyses are intended to characterize longitudinal patterns in participant-reported outcomes and clinical variables following elafibranor initiation in routine clinical practice. No formal hypothesis testing is planned unless otherwise prespecified in the SAP. Continuous variables will be summarized using descriptive statistics, including mean, standard deviation, median, minimum, maximum and 95% confidence intervals, as appropriate. Counts and percentages will be used for categorical variables. Missing data will not be routinely imputed. Exploratory analyses may include correlation analyses and regression modelling, as appropriate, to evaluate associations between liver function parameters and participant-reported outcomes. Results of exploratory analyses will be interpreted descriptively and not as confirmatory evidence.