NN7088-4029 A multinational, prospective, open labelled, non-controlled, non-interventional post-authorisation study of turoctocog alfa pegol (N8-GP) during long-term routine prophylaxis and treatment of bleeding episodes in patients with haemophilia A (pathfinder 9)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Safety 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:
  TUROCTOCOG ALFA PEGOL
• Medical condition to be studied:
  Factor VIII deficiency

Population studied

• 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)
• Estimated number of subjects:
  60

Study design details

• Main study objective:
  The primary objective of the study is to investigate the safety of N8-GP including the PEG moiety during prophylaxis and long-term use in patients with haemophilia A as prescribed by the physician.
• Outcomes:
  Number of Adverse Events (AEs) reported during the study period from inclusion of the patient until end of study, Number of Serious Adverse Events (SAEs) reported during the study period from inclusion of the patient until end of study
• Data analysis plan:
  No formal testing of statistical hypothesis will be performed. All data will be presented using descriptive statistics. Categorial data will be summarized by frequency tables while continuous data will be summarized by mean, standard deviation, median, minimum and maximum value. Subgroup analysis will be presented: - By age groups (< 6 years, 6 to < 12 years, 12 to < 18 years, 18 to < 65 years, ≥ 65 years) - By severity of disease (moderate and severe) Patients who previously developed inhibitors before entering this study might be presented separately if deemed necessary.

Documents

Study report

4029 progress report eu-pas-reg 01 redacted

4029 progress report eu-pas-reg 02 redacted

Study, other information

4029 progress report eu-pas-reg 02 redacted.pdf