TAK-669-4018: A Survey Among Patients, Caregivers and Home Infusion Nurses Based in the European Union to Assess Their Awareness and Understanding of Educational Materials Supporting VPRIV® Infusion at Home

Study type

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

  Other
• If ‘other’, further details on the scope of the study:
  Assessing awareness and understanding of education materials for using VPRIV®
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Cross-sectional

Study drug and medical condition

• Medicinal product name:
  VPRIV
• Study drug International non-proprietary name (INN) or common name:
  VELAGLUCERASE ALFA
• Anatomical Therapeutic Chemical (ATC) code:
  (A16AB10) velaglucerase alfa

  velaglucerase alfa
• Medical condition to be studied:
  Gaucher's disease

Population studied

• Short description of the study population:
  Patients, Caregivers and Home Infusion Nurses Based in the European Union
• Age groups:
  • 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 main objective of this study is to assess the proportion of participants, caregivers and home infusion nurses who are aware of the Educational Material (EM) (home infusion guide, infusion diary, and safety and emergency plan), who understand the EM and use the EM.
• Outcomes:
  1. Percentage of Participants/Caregivers who are Aware of EM 2. Percentage of Participants/Caregivers who Understand EM 3. Percentage of Participants/Caregivers who Utilize EM 4. Percentage of Home Infusion Nurses Aware of EM 5. Percentage of Home Infusion Nurses who Understand EM 6. Percentage of Home Infusion Nurses who Utilize EM
• Data analysis plan:
  Percentage of correct and appropriate answers about awareness and understanding of conditions of use of VPRIV® home infusion given by patients, caregivers and home infusion nurses will be assessed. Continuous variables will be described by the number of valid cases and missing data, mean, standard deviation (SD), median, Q1, Q3, minimum, and maximum. No missing data will be replaced. Categorical variables will be described as the total number and relative percentage per category. CIs of 95% will be calculated when relevant.