Eptacog Beta post marketing safety surveillance using the PedNet registry (F7TG2207 – PedNet Registry)

Study type

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

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Post marketing safety study based on PedNed Registry

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  EPTACOG BETA (ACTIVATED)
• Medical condition to be studied:
  Factor VIII deficiency

  Factor IX deficiency
• Additional medical condition(s):
  with inhibitors

Population studied

• Age groups:
  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)

  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:
  Renal impaired

  Hepatic impaired

  Immunocompromised

  Pregnant women
• Estimated number of subjects:
  15

Study design details

• Main study objective:
  The primary objective will be to determine the number and incidence of important potential risks defined as: - Hypersensitivity reactions (including anaphylactic reactions) - Thromboembolic events (including those due to drug-drug interactions with activated or nonactivated prothrombin complex or other haemostatic agents) - Immunogenicity - Death
• Outcomes:
  - Number of allergic reactions, thromboembolic events, and unexpected poor efficacy secondary to immunogenicity - Incidence of allergic reactions, thromboembolic events, and unexpected poor efficacy secondary to immunogenicity - Incidence of antidrug antibodies
• Data analysis plan:
  Data management is under the responsibility of the PedNet Registry according to the current version of the PedNet Protocol. Data are collected by centres in the PedNet registry and monitored to improve data quality. The study duration will be 5 years with a detailed annual report provided by the PedNet registry after each full year of surveillance. The number and incidence of subjects with Treatment-emergent AEs (TEAEs), specifically allergic, thromboembolic events and unexpected poor efficacy secondary to immunogenicity will be assessed and presented in a final report.