EUropean REgistry in Children below six years of age treated with BeneFIX (EUREKIX)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Other

  Safety study (incl. comparative)
• If ‘other’, further details on the scope of the study:
  Efficacy of BeneFIX in children below 6 years of age
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  This is a two phase, non-interventional, multicenter trial including a retrospective (Phase I) and/or prospective (Phase II) data collection period.

Study drug and medical condition

• Medical condition to be studied:
  Haemophilia B with anti factor IX

Population studied

• Short description of the study population:
  Patients were eligible to take part in the retrospective data collection if they have been treated with BeneFIX for at least 12 months at an age below 6 years and are at time of consent not older than 8 years. Patients were eligible to participate in the prospective part of the study if they are able to accrue at least 12 months of data in the study before reaching the age of 6 years. The maximum time of prospective observation is 24 months.
• Age groups:
  • Children (2 to < 12 years)
• Special population of interest:
  Other
• Special population of interest, other:
  Haemophilia B patients
• Estimated number of subjects:
  50

Study design details

• Main study objective:
  The objective of the study is to collect data in Europe regarding safety (primary endpoint) and efficacy (secondary endpoint) of treatment with rFIX (BeneFIX®) in children below 6 years of age treated in the routine clinical setting
• Data analysis plan:
  The efficacy of BeneFIX® will be descriptively assessed by different measurement parameters: –Annualized bleeding rates (ABRs) –Responses to the on-demand and prophylactic treatment with BeneFIX, respectively, for all bleeds and according to bleeding location (4-point scale of assessment: excellent, good, moderate, no response) –The incidence of less-than-expected therapeutic effect (LETE) will be assessed by the investigator using the criteria listed in Section 8.7.2.3. –Lack of effect, defined as the failure of expected pharmacologic action or therapeutic benefitIn the event of a bleed in the on-demand setting (including those occurring during the prophylaxis period), the 4-point response scale for an on-demand treatment of a bleeding episode is defined as follows: •Excellent•Good•Moderate•No Response