TAK-577-4005: Estimating Risk of Selected Adverse Events in Patients with Von Willebrand Disease Treated With VEYVONDI® (Vonicog Alfa; Recombinant Von Willebrand Factor)

Study type

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

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medical condition to be studied:
  Von Willebrand's disease

Population studied

• Age groups:
  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:
  80

Study design details

• Main study objective:
  To estimate risk of hypersensitivity reactions, thromboembolic events, and VWF or FVIII inhibitor formation after treatment with VEYVONDI in study population for treatment of haemorrhage, surgical bleeding and prevention of surgical bleeding when DDAVP treatment alone is ineffective or not indicated and describe association of thromboembolic events with use of FVIII.
• Outcomes:
  The primary outcomes will assess the percentage of participants who experienced hypersensitivity reactions, thromboembolic events, and with VWF or factor VIII (FVIII) inhibitor formation.
• Data analysis plan:
  Descriptive statistics will be generated within each data source to describe study population. Categorical variables will be summarized by frequencies and proportions, and continuous variables will be summarized as the mean with standard deviation (SD) or standard error (SE) and range for normally-distributed variables, median, interquartile range (IQR) and range for non-normally-distributed variables.

Documents

Study report

TAK-577-4005-clinical-study-report-redact.pdf