A Low-Interventional Multicentre Post-Authorisation Safety Study for Voncento / Biostate / Aleviate for Routine Prophylaxis, Treatment of Bleeding Events and / or Surgery in Male Subjects with Haemophilia A (Biostate_4001)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness
• Data collection methods:
  Combined primary data collection and secondary use of data

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Low interventional, Post-Authorisation Safety Study

Study drug and medical condition

• Name of medicine:
  VONCENTO
• Name of medicine, other:
  Biostate, Aleviate
• Study drug International non-proprietary name (INN) or common name:
  HUMAN COAGULATION FACTOR VIII
• Anatomical Therapeutic Chemical (ATC) code:
  (B02BD06) von Willebrand factor and coagulation factor VIII in combination

  von Willebrand factor and coagulation factor VIII in combination
• Medical condition to be studied:
  Factor VIII deficiency

Population studied

• Short description of the study population:
  Subjects with haemophilia A who have been treated with Voncento for the treatment of acute bleeding episodes, routine prophylaxis, and / or perioperative bleeding.
• Age groups:
  • Paediatric Population (< 18 years)

    • Infants and toddlers (28 days – 23 months)
    • Children (2 to < 12 years)
    • Adolescents (12 to < 18 years)
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 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:
  100

Study design details

• Main study objective:
  To investigate the immunogenicity of Voncento in Previously Treated Patients (PTPs) with haemophilia A.
• Outcomes:
  The incidence of FVIII inhibitor development, Nature and incidence of reported adverse events, serious adverse events, and adverse events of special interest, and lack of effect (ie, a less than expected response to Voncento treatment when given for a bleeding event) as assessed by the investigator.
• Data analysis plan:
  All analyses will be performed for the Safety Population, which will comprise all enrolled subjects who received at least 1 dose of Voncento after signed consent to participate in the study. Study endpoints will be summarised by descriptive statistics. Continuous variables will be summarised in terms of the mean, standard deviation, median, minimum, and maximum. Other descriptive statistics (eg, quartiles, coefficients of variation) may be reported when appropriate. Categorical variables will be summarised using frequency counts and percentages. Confidence intervals will be calculated for key parameters or estimates as warranted.