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 topic, other:
  Observational study
• 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

• 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:
  The registry population will comprise participants with PFIC (all types) enrolled into the Ipsen odevixibat PFIC registry. Participants with PFIC who have been prescribed odevixibat by their treating physician will be eligible
• 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)
• Estimated number of subjects:
  10

Study design details

• Study design:
  This study will collect information from people with Progressive Familial Intrahepatic Cholestasis (PFIC) as they use odevixibat in their daily lives.
• Main study objective:
  To evaluate the long-term safety of odevixibat based on adverse events (AEs).
• Outcomes:
  Primary Outcome Measure:; 1.Percentage of participants experiencing Adverse Events (AEs); 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.; [Time Frame: From first ICF signature and up to end of data collection (approximately 7 years of data collection)]; Secondary Outcome Measures:; 2. Event-free survival (EFS); EFS is defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion, liver transplant, or death; [Time Frame: From first ICF signature and up to end of data collection (approximately 7 years of data collection)]; 3. Surgical biliary diversion-free survival; Surgical biliary diversion-free survival is defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion or death; [Time Frame: From first ICF signature and up to end of data collection (approximately 7 years of data collection)]; 4. Liver transplant-free survival; Liver transplant-free survival is defined as time from the start of odevixibat treatment to the first occurrence of liver transplant or death; [Time Frame: From first ICF signature and up to end of data collection (approximately 7 years of data collection)]; 5. Overall survival (OS); OS is defined as the time from the start of odevixibat treatment to death; [Time Frame: From first ICF signature and up to end of data collection (approximately 7 years of data collection)]
• Data analysis plan:
  No formal sample size calculations have been performed for this registry. Enrolment for this registry will be based on the number of participants prescribed odevixibat and their willingness to participate in the registry, but the goal will be to enrol a minimum of 10 participants with PFIC (all types) in South Korea