Registry to Document Treatment Effectiveness, Safety, Including Prospective Long-term Outcomes in Participants with Progressive Familial Intrahepatic Cholestasis (PFIC) who take Odevixibat (Bylvay)

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Effectiveness study (incl. comparative)

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

  odevixibat
• Medical condition to be studied:
  Progressive familial intrahepatic cholestasis

Population studied

• Short description of the study population:
  Participants with Progressive Familial Intrahepatic Cholestasis (PFIC) (all types) who have been prescribed odevixibat by their treating physician will be eligible. Participants who started odevixibat treatment before the implementation of the registry may also be enrolled.
• 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:
  20

Study design details

• Study design:
  This will be a long-term, observational, prospective, and voluntary participation registry-based study designed to examine the real-world usage of odevixibat for the treatment of PFIC.
• Main study objective:
  The primary objective of this registry-based study is to evaluate the long-term safety of odevixibat based on adverse events (AEs).
• Outcomes:
  Safety Endpoints:; • All AEs, according to incidence, severity grade, causality, outcome, action taken, and seriousness; ; 7.3.1.2 Effectiveness Endpoints; • Event-free survival (EFS), defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion, liver transplant, or death; • Surgical biliary diversion-free survival, defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion or death; • Liver transplant-free survival defined as time from the start of odevixibat treatment to the first occurrence of liver transplant or death; • Overall survival, defined as the time from the start of odevixibat treatment to death; • Pruritus improvement, described at each patient visit using a (semi-) objective scoring scale to assess the level of pruritus from the start of the odevixibat treatment; • Change from baseline in serum bile acid, assessed by measuring serum bile acid levels at each patient visit
• Data analysis plan:
  No formal sample size calculations have been performed for this registry-based study.; Enrolment for this registry-based study will be based on the number of participants prescribed odevixibat, but the goal will be to enrol a minimum of 20 participants with PFIC (all types) in China.