091301: FEIBA NF GLOBAL OUTCOME STUDY (FEIBA-GO) (091301: FEIBA-GO)

Study type

• Study topic:
  Human medicinal product

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

  Effectiveness study (incl. comparative)

  Safety study (incl. comparative)

  Other
• If ‘other’, further details on the scope of the study:
  Pharmacodynamic study, Health-Related Quality of Life Analysis
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Cohort

  Other
• Non-interventional study design, other:
  Post-authorization, prospective, uncontrolled, observational, non-interventional, open-label, multicenter cohort study

Study drug and medical condition

• Name of medicine, other:
  FEIBA NF
• Additional medical condition(s):
  Haemophilia A or B with high responding inhibitors

Population studied

• Short description of the study population:
  Patients were male hemophilia A or B patients with high-responding inhibitors who had been prescribed FEIBA for the treatment or prevention of bleeding events by a treating physician prior to the decision to enroll in the study. No additional diagnostic or monitoring procedures were applied to patients, except those that were part of normal/routine clinical practice.
• Age groups:
  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:
  Other
• Special population of interest, other:
  Hemophilia A or B patients
• Estimated number of subjects:
  55

Study design details

• Main study objective:
  The primary objective of the study is to describe the hemostatic effectiveness of FEIBA NF in a variety of clinical settings including on-demand therapy, prophylaxis and immune tolerance induction (ITI) in haemophilia A or B patients with high-responding inhibitors.
• Outcomes:
  (A) 1) Prophylaxis: Treatment of breakthrough bleeds 2) On-demand: Treatment of BEs: -Bleed rates -Types of bleeding events -Number of BEs with corresponding hemostatic efficacy ratings(B) 1) Prophylaxis: Totals & breakthrough bleeds 2) On-demand: -Number of infusions- Weight adjusted dose- Total units of FEIBA NF infused, 1) Joint clinical outcomes in routine clinical practice setting, using any therapeutic regimen, assessed as in common practice2) Health-Related Quality of Life using standardized assessment questionnaires/ tools
• Data analysis plan:
  Descriptive statistics will include specifically but not exclusively, arithmetic mean, standard deviations, medians, minimum, maximum, 25th and 75th percentiles, proportions, frequency counts and 95% confidence intervals of select point estimates. Figures will be prepared to illustrate the patterns of data over time where appropriate.

Documents

Study results

091301-clinical-study-report-redact