Prospective Non-Interventional Study Evaluating the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS)

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:
  Case-only

Study drug and medical condition

• Medicinal product name:
  KAYFANDA
• Study drug International non-proprietary name (INN) or common name:
  ODEVIXIBAT
• Anatomical Therapeutic Chemical (ATC) code:
  (A05AX05) odevixibat

  odevixibat
• Medical condition to be studied:
  Alagille syndrome

Population studied

• Short description of the study population:
  Participants with Alagille syndrome (ALGS) who have been prescribed odevixibat by their treating physician will be enrolled into the study. Participants who started odevixibat treatment before study implementation may also be enrolled. The study will be conducted in the EU/EEA.
• Age groups:
  • In utero
  • Paediatric Population (< 18 years)

    • 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)
• Estimated number of subjects:
  30

Study design details

• Study design:
  This study will collect information from patients with ALGS who are using odevixibat in their daily lives. Odevixibat is a medication that helps patients with ALGS, a rare disease that affects the liver and causes itching.
• Main study objective:
  To evaluate the incidence of adverse events (AEs) and serious adverse events (SAEs) in participants with ALGS chronically treated with odevixibat.
• Outcomes:
  Primary outcomes are the percentage of participants experiencing adverse events (AEs) and serious adverse events (SAEs). ; Secondary outcomes include diarrhoea-related events, fat-soluble vitamin deficiencies and related manifestations, suspected hepatotoxicity and related manifestations, changes from baseline in ALT, AST, GGT, blood bilirubin and INR, hospitalisations and treatment discontinuations related to diarrhoea, hepatotoxicity or fat-soluble vitamin deficiency, pregnancy and maternal complications, adverse effects in the foetus/neonate/infant after exposure during pregnancy or lactation, biliary diversion surgery, liver transplantation, death, and switching from odevixibat to maralixibat.
• Data analysis plan:
  No formal sample size calculation was performed. Enrolment will be based on the number of participants prescribed odevixibat and their willingness to participate in the study, but the goal will be to enrol approximately 30 participants with ALGS in the EU/EEA based on current estimates.