A POST AUTHORIZATION SAFETY SURVEILLANCE REGISTRY IN PREVIOUSLY UNTREATED PATIENTS WITH SEVERE HEMOPHILIA A IN USUAL CARE SETTINGS

Study type

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

  Effectiveness study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Primary data collection, voluntary prospective Registry

Study drug and medical condition

• Name of medicine:
  REFACTO AF
• Additional medical condition(s):
  SEVERE HEMOPHILIA A

Population studied

• Age groups:
  • 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)
• Estimated number of subjects:
  100

Study design details

• Main study objective:
  The primary objective of this Registry is to evaluate clinically significant FVIII inhibitor development in hemophilia A subjects who were PUPs and ≤6 years of age upon initiating treatment with ReFacto AF (first group) as well as in hemophilia A subjects who had attained 50 to 149 EDs, are ≤8 years of age, and successfully completed participation in another study (B1831006) (second group).
• Outcomes:
  Clinically significant positive FVIII inhibitor, which is defined in this Registry as:a. 2 positive FVIII inhibitor results (using either Bethesda Inhibitor Assay or the Nijmegen Modification of the Bethesda Inhibitor Assay) within a 4 week period. Occurrence of adverse events (AEs).
• Data analysis plan:
  The results of this study will be presented using descriptive statistics. The primary analysis will be performed on all subjects who receive at least one dose of ReFacto AF during participation in this Registry. The two groups: prospectively identified PUPs (with 0 to 7 ReFacto AF EDs at the time of Registry enrollment, Group A subjects), and subjects who completed study B1831006 (with at least 50 EDs and “completed” B1831006 and not “withdrawn or discontinued”) will be analyzed independently with respect to hemophilia history, adverse events, inhibitor development, demography, and disposition. The primary safety outcome, the proportion of subjects who develop clinically significant inhibitors, will be reported. Secondary safety outcomes, the frequency of adverse events and serious adverse events, will be summarized. The reasons for discontinuation from the Registry or from treatment with ReFacto AF therapy will be described.