ClinicalTrials.gov record NCT05669729
A Survey to Assess Participants', Caregivers', and Nurses' Use and Understanding of Educational Material on Velaglucerase Alfa (VPRIV) Home Infusion
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
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This observational survey asks people with Gaucher disease who receive VPRIV at home, their caregivers, and home‑infusion nurses about their awareness, understanding, and use of VPRIV educational materials. Participants complete a non‑nominative web‑ or paper questionnaire that takes about 10–15 minutes. The study is cross‑sectional and collects responses from a single group on one day; target enrollment is about 30 participants/caregivers and 30 nurses.
What it is studying. Whether patients receiving VPRIV home infusions, their caregivers, and home infusion nurses are aware of, understand, and use the educational materials provided for VPRIV home infusion (the home infusion guide, infusion diary, and safety/emergency plan).
What the researchers hope to learn. To determine the percentages of patients, caregivers, and nurses who are aware of, who understand, and who use the VPRIV educational materials (home infusion guide, infusion diary, safety/emergency plan).
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Gaucher disease participants who receive VPRIV® for home infusion and their caregivers.
- 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).
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
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.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Take a one-time, non‑nominative questionnaire (web-based or paper) about awareness, understanding, and use of VPRIV® educational materials (home infusion guide, infusion diary, emergency plan). The survey is completed on Day 1 and is expected to take about 10–15 minutes. Responses are collected from patients, caregivers, or nurses. The registry describes only this single questionnaire; no invasive procedures are listed.
How the study is structured. Single‑group, cross‑sectional observational survey. Each participant (patient, caregiver, or nurse) completes one anonymous web‑ or paper questionnaire (about 10–15 minutes). Responses are collected once (Day 1); planned sample ~30 patients/caregivers and ~30 nurses.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
- The registry says recruitment has not started. When is it expected to open, and can I register interest now?
- No sites are listed on the registry yet. Where will the study take place?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on Gaucher disease
Important dates
- Study start
- Jan 31, 2027 (estimated)
- Primary completion
- Jun 30, 2027 (estimated)
- Study completion
- Sep 30, 2027 (estimated)
- Target enrollment
- 60 participants (estimated)
- Last updated on registry
- Oct 1, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Takeda
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Takeda Contact 📞 +1-877-825-3327 ✉ medinfoUS@takeda.com
Participating sites
Study locations have not been posted on ClinicalTrials.gov yet. This study is not yet recruiting, so sites may be added when recruitment begins. The central study contact above may be able to tell you about planned sites.
Terms used on this page · explanations by MedicalUpdates.ai
- Inclusion criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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