Surveillance of Safety and Efficacy of wilate® in patients with von Willebrand disease (Wil-20)

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:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Observational post-marketing surveillance

Study drug and medical condition

• Name of medicine, other:
  Wilate - B02BD06
• Medical condition to be studied:
  Von Willebrand's disease

Population studied

• Short description of the study population:
  von Willebrand disease (VWD) patients of any gender, age, or VWD type, previously treated (PTPs) or previously untreated patients (PUPs).
• 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)
• Special population of interest:
  Other
• Special population of interest, other:
  von Willebrand disease (VWD) patients
• Estimated number of subjects:
  50

Study design details

• Main study objective:
  Primary objective is to document the safety and tolerability of wilate® for prophylaxis and treatment of bleeding in VWD, incl. surgeriesSecondary objective:Secondary objective is to document the efficacy of wilate® in the treatment of acute bleeding, in the prophylaxis of VWD and in interventional procedures (e.g. minor/major surgery, dental care, invasive diagnostic procedures etc.).
• Outcomes:
  Assessment of safety will be based on recorded Adverse Drug Reactions during the full course of the observation. Assessment of tolerability will be based on a 3 point Verbal Rating Scale. Assessment of efficacy of wilate® in prevention and/or treatment of bleeding episodes and in surgical procedures will be based on a 4-point hemostatic efficacy scale as “excellent”, “good” “moderate” or “none”.
• Data analysis plan:
  The responsibility for the statistical analyses presented in the final report belongs to: contract research organisation: GASD, Gesellschaft für Angewandte Statistik + Datenanalyse mbH, Am Konvent 8 - 10, 41460 Neuss, Germany.This is a prospective post-licensure surveillance that will be conducted as an international multi-centre non-interventional surveillance. All items of the CRF will be analyzed by means of descriptive statistical methods.