NN7999-4031: A Non-Interventional Post-Authorisation Safety Study (PASS) in male haemophilia B patients receiving Nonacog Beta Pegol (N9-GP) prophylaxis treatment

Study type

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

  Drug utilisation

  Effectiveness study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  NONACOG BETA PEGOL
• Medical condition to be studied:
  Haemophilia B without 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)
• Estimated number of subjects:
  70

Study design details

• Main study objective:
  The primary objective of the study is to investigate the safety of N9-GP in prophylaxis and during long-term routine use (Adverse Drug Reactions) in patients with haemophilia B in the manner it isprescribed by physicians. This will include assessment of specific pharmacological risks for FIX replacement products (FIX inhibitors, allergic reactions, and thromboembolic events).
• Outcomes:
  Number of Adverse Drug Reactions (ADRs) (FIX inhibitors, allergic reactions, and thromboembolic events) reported during the study period, No.of SAEs.-No.of bleeding episodes,long-term routine N9-GP use assessed by ABR.-No.of treatment requiring bleeding episodes,long-term routine N9-GP use,ABR assessed.-Haemostatic effect of N9-GP for treatment assessed by success/failure on a 4-point scale.-Haemostatic response of N9-GP in perioperative management assessed as success/failure on a 4-point scale.-All reported for up to 9 years
• Data analysis plan:
  No formal testing of statistical hypotheses will be performed. Evaluation of data will be based upon descriptive statistics, i.e. summary tables, listings and figures. Categorical data will be summarised by frequency tables while continuous data will be summarized by mean, standard deviation, median, minimum and maximum value separated into age groups.