Prospective, multinational, non-interventional post-authorisation study to document the long-term immunogenicity, safety, and efficacy of Human-cl rhFVIII (simoctocog alfa) in patients with haemophilia A treated in routine clinical practice (GENA-99)

Study type

• Study topic:
  Human medicinal product

  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Other
• If ‘other’, further details on the scope of the study:
  To collect clinical data to ensure the consistency, in the long-term, between the outcome from pre-authorisation clinical studies (in 135 previously treated patients)
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Cohort

  Other
• Non-interventional study design, other:
  Non randomised observational study, Post-authorisation study

Study drug and medical condition

• Anatomical Therapeutic Chemical (ATC) code:
  (B02BD02) coagulation factor VIII

  coagulation factor VIII
• Medical condition to be studied:
  Haemophilia A without inhibitors

Population studied

• Short description of the study population:
  Male patients with haemophilia A without inhibitors treated in routine clinical practice receiving rhFVIII (simoctocog alfa); At least 100 patients should have severe haemophilia A. Approx. 60 patients should be < 12 years of age; also, at least 10 patients should be aged between 14–18 years. Patients with severe haemophilia A after successful immune tolerance induction (ITI) can also be included; the proportion of ITI patients should not exceed 25% of the entire cohort.
• Age groups:
  • 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 patients
• Estimated number of subjects:
  200

Study design details

• Main study objective:
  •To assess the long-term immunogenicity and safety of Human-cl rhFVIII in treating or preventing bleeding episodes (BEs) in patients with haemophilia A• To assess the long-term efficacy of Human-cl rhFVIII in treating or preventing BEs in patients with haemophilia A
• Outcomes:
  long-term immunogenicity and safety of Human-cl rhFVIII,long-term efficacy of Human-cl rhFVIII
• Data analysis plan:
  The data recorded in the CRF will be analysed using descriptive statistical methods. An analysis plan detailing the analyses to be performed will be prepared by the study statistician and approved by the Sponsor before the start of the study.