Elafibranor Pregnancy Surveillance Program: A Study to Evaluate the Safety of Elafibranor During Pregnancy

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Safety study (incl. comparative)
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• 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:
  Pregnancy

Population studied

• Short description of the study population:
  The participants of this study will be of any age who are exposed to at least one dose of elafibranor at any time during pregnancy, or since 3 weeks prior to the conception (based on estimated last menstrual period [LMP])
• Age groups:
  • In utero
  • Paediatric Population (< 18 years)

    • Neonate

      • Preterm newborn infants (0 – 27 days)
      • Term newborn infants (0 – 27 days)
    • Infants and toddlers (28 days – 23 months)
    • Children (2 to < 12 years)
    • Adolescents (12 to < 18 years)
  • 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:
  Pregnant women
• Special population of interest, other:
  infant
• Estimated number of subjects:
  3

Study design details

• Study design:
  The study begins once the first participant is enrolled and ends after the last mother and child data are collected. It is planned to run for about 10 years, with infant follow‑up lasting up to 2 years, for a maximum total duration of 12 years and 9 months.
• Main study objective:
  To describe the occurrence of congenital malformations and developmental delays in the offspring of participants exposed to elafibranor during pregnancy.
• Comparators:
  To contextualize the surveillance program data, outcomes will be compared to those of participants with primary biliary cholangitis (PBC) who became pregnant after their PBC diagnosis but were not exposed to elafibranor, using publicly available literature, as well as considering general population data.
• Outcomes:
  Primary Safety Endpoint: ; • Prevalence of congenital malformations.; ; Secondary Safety Endpoints:; • Prevalence of minor and major congenital malformations; • Prevalence of molar/ectopic pregnancy; • Prevalence of fetal loss, including SAB, stillbirths, and terminations (medically indicated or elective or medically indicated abortion); • Prevalence of live births; • Prevalence of premature delivery; • Prevalence of infants born SGA; • Prevalence of neonatal/infant death; • Prevalence of postnatal growth deficiency at 4 months, 12 months and 24 months; • Prevalence of infant developmental delay at 4 months, 12 months and 24 months; • Prevalence of infant healthcare requirements and interventions not considered standard, including but not limited to:; - Infant hospitalization due to serious illness; - Emergency department visits; - Specialist or other healthcare professional consultations; - Developmental assessments and interventions; - Therapeutic services; - Educational support and adaptations; • Incidence and nature of all adverse events (AEs); • Changes in biochemical markers of cholestasis from last available measurement before pregnancy and through pregnancy and postpartum:; - ALP; - Bilirubin; - ALT; - AST; - GGT; - Albumin; - Bile acids; - Creatine phosphokinase (CPK); - Lipid profile (including total cholesterol, LDL-C, HDL-C, VLDL-C and triglycerides)
• Data analysis plan:
  All the analyses will be primarily descriptive in nature. For each continuous variable, the number of observations, median, mean, standard deviation, interquartile range, minimum and maximum will be reported. For each categorical variable, the frequency and percentage in each category will be reported. The frequency and percentage of subjects with missing data for each data point will be presented. Results will be rounded off to one decimal place; therefore, percentages may not always add up to 100.; ; The prevalence of each outcome will be calculated by dividing the number of cases of the outcome by the appropriate denominator for that particular outcome.; ; The study will include planned interim clinical reports, with the first to be drafted by the end of September 2030.