Clinical trial NCT05669729: A Survey to Assess Participants', Caregivers', and Nurses' Use and Understanding of Educational Material on Velaglucerase Alfa (VPRIV) Home Infusion
Official title: 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: OBSERVATIONAL
Overall status: NOT_YET_RECRUITING
Conditions: Gaucher Disease
Keywords: Drug Therapy
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2023-01-03
Enrollment: 60
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: This study will recruit Gaucher disease participants receiving VPRIV® home infusion, their caregivers and home infusion nurses who administer VPRIV® infusion at home in European countries.

Brief summary: The main purpose of this survey is to determine participants', caregivers', and nurses' understanding and use of educational materials (EM) on VPRIV home treatment. EM includes an infusion diary and guide and an emergency plan related to VPRIV infusion given at home for Gaucher disease. The survey is conducted in European countries.

Data will be collected directly from participants, caregivers, and nurses in form of a questionnaire, electronic or paper.

Detailed description: The participants diagnosed with Gaucher disease receiving VPRIV® home infusion, their caregivers and home infusion nurses were observed in this study. This cross-sectional study will determine and assess the understanding of the educational material (EM), including infusion diary and emergency plan associated with VPRIV® home infusion in participants diagnosed with Gaucher disease, their caregivers and home infusion nurses.

The study will recruit approximately 30 participants/caregivers and 30 home infusion nurses. The survey will be conducted through non-nominative web-based questionnaires that will be completed in approximately 10-15 minutes. All participants will be enrolled in a single observational group:

• VPRIV® Home Infusion

The trial will be conducted in home-based setting in Austria, Belgium, Finland, France, Germany, Italy, the Netherlands, and Spain.

Eligibility criteria: Inclusion criteria:

1. Gaucher disease participants who receive VPRIV® for home infusion and their caregivers.
2. Home infusion nurses administering VPRIV® to participants with Gaucher disease at home.

Exclusion criteria:

Potential respondents will be excluded if they have conflicts of interest with the survey (e.g., if they are employed by regulatory bodies, or pharmaceutical industry).

Primary outcomes:
measure: Percentage of Participants and Caregivers Aware of Educational Material (EM)
timeFrame: Day 1
measure: Percentage of Participants and Caregivers who Understand the EM
timeFrame: Day 1
measure: Percentage of Participants and Caregivers who Utilize the EM
timeFrame: Day 1
measure: Percentage of Home Infusion Nurses Aware of the EM
timeFrame: Day 1
measure: Percentage of Home Infusion Nurses who Understand the EM
timeFrame: Day 1
measure: Percentage of Home Infusion Nurses who Utilize the EM
timeFrame: Day 1
Secondary outcomes:
Other outcomes:
measure: Usefulness of the EM for Participants and Caregivers
timeFrame: Day 1
measure: Usefulness of the EM for Home Infusion Nurses
timeFrame: Day 1
Locations:
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: