Prospective Registry-Based Study of the Long-Term Safety of Odevixibat in Patients with Progressive Familial Intrahepatic Cholestasis (PFIC)

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  BYLVAY
• Name of medicine, other:
  Odevixibat
• Anatomical Therapeutic Chemical (ATC) code:
  (A05AX05) odevixibat

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

Population studied

• Age groups:
  Infants and toddlers (28 days – 23 months)

  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)

  Adults (18 to < 46 years)

  Adults (46 to < 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:
  100

Study design details

• Main study objective:
  To assess the long-term, real-world safety profile of odevixibat treatment in patients with PFIC compared to patients not receiving odevixibat (untreated control cohort).
• Outcomes:
  Yes: Incidence of: diarrhoea, clinical manifestations of FSV deficiency, change in FSV levels, reports of ineffectiveness of previously effective fat-soluble drugs, new nutritional interventions, clinical manifestations of hepatotoxicity, hospitalizations or discontinuation of treatment due to diarrhoea, FSV deficiency or hepatotoxicity. Changes in ALT, AST bilirubin, INR or growth parameters.
• Data analysis plan:
  Descriptive analysis will be conducted and presented by odevixibat cohort (Patients with PFIC who received odevixibat at any time before or during the study) and control cohort (Patients with PFIC who did not receive odevixibat). Demographic and baseline characteristics of all patients will be described by cohort using mean, standard deviation, median, minimum and maximum for continuous variables and count and percentages for discrete variables. For the safety endpoints, the number of events and incident rates will be calculated. AEs will also be analysed by incident users and prevalent users separately as a part of a subgroup analysis. For clinical laboratory variables, descriptive statistics for results and change from baseline at each follow-up visit (year) will be presented for each cohort.

Documents

Study report

A4250-019_Synopsis_Redacted_PDFA (1).pdf