Gaucher DiseaseNCT05669729
A Survey to Assess Participants', Caregivers', and Nurses' Use and Understanding of Educational Material on Velaglucerase Alfa (VPRIV) Home Infusion
An observational study of Gaucher Disease, run by Takeda.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Gaucher Disease
- Type of treatment
- Injection or infusion
- How many people
- 60 participants
- Where
- No sites listed yet
- Study dates
- 31 Jan 2027to 30 Sep 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- Gaucher disease participants who receive VPRIV® for home infusion and their caregivers.
- Home infusion nurses administering VPRIV® to participants with Gaucher disease at home.
Who cannot join
- 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).
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
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
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.
Read the 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.
- Conditions
- Gaucher Disease
- Keywords
- Drug Therapy
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| VPRIV® Home Infusion | Participants diagnosed with Gaucher disease will receive VPRIV® home infusion, administered by their caregivers and home infusion nurses will participate in the survey and complete the non-nominative web-based questionnaires on Day 1. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants and Caregivers Aware of Educational Material (EM)
EM includes home infusion guide, infusion diary, and safety and emergency plan.
Measured: Day 1
Percentage of Participants and Caregivers who Understand the EM
EM includes home infusion guide, infusion diary, and safety and emergency plan.
Measured: Day 1
Percentage of Participants and Caregivers who Utilize the EM
EM includes home infusion guide, infusion diary, and safety and emergency plan.
Measured: Day 1
Percentage of Home Infusion Nurses Aware of the EM
EM includes home infusion guide, infusion diary, and safety and emergency plan.
Measured: Day 1
Percentage of Home Infusion Nurses who Understand the EM
EM includes home infusion guide, infusion diary, and safety and emergency plan.
Measured: Day 1
Percentage of Home Infusion Nurses who Utilize the EM
EM includes home infusion guide, infusion diary, and safety and emergency plan.
Measured: Day 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jan 2023
- Study start31 Jan 2027 (estimated)
- Primary completion30 Jun 2027 (estimated)
- Study completion30 Sep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gaucher DiseaseStudies of the same condition, open and under way.
- More trials run by TakedaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Takeda
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05669729
- Sponsor's protocol ID
- TAK-669-4018
- Status last verified
- Sep 2026
- First submitted
- 20 Dec 2022
- Last updated
- 1 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Study at a glance
From the registry record, last updated by the sponsor on 1 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05669729
- Acronym / sponsor ID
- TAK-669-4018
- Conditions
- Gaucher Disease
- Keywords
- Drug Therapy
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- TakedaIndustry (pharma, biotech, devices)
- Study start
- 31 Jan 2027 (estimated)
- Primary completion
- 30 Jun 2027 (estimated)
- Study completion
- 30 Sep 2027 (estimated)
- First posted
- 3 Jan 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| VPRIV® Home Infusion | – | –Participants diagnosed with Gaucher disease will receive VPRIV® home infusion, administered by their caregivers and home infusion nurses will participate in the survey and complete the non-nominative web-based questionnaires on Day 1. |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (2 listed)
- Gaucher disease participants who receive VPRIV® for home infusion and their caregivers.
- Home infusion nurses administering VPRIV® to participants with Gaucher disease at home.
Key exclusion criteria (1 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Percentage of Participants and Caregivers Aware of Educational Material (EM)
Time frame: Day 1
EM includes home infusion guide, infusion diary, and safety and emergency plan.
Percentage of Participants and Caregivers who Understand the EM
Time frame: Day 1
EM includes home infusion guide, infusion diary, and safety and emergency plan.
Percentage of Participants and Caregivers who Utilize the EM
Time frame: Day 1
EM includes home infusion guide, infusion diary, and safety and emergency plan.
Percentage of Home Infusion Nurses Aware of the EM
Time frame: Day 1
EM includes home infusion guide, infusion diary, and safety and emergency plan.
Percentage of Home Infusion Nurses who Understand the EM
Time frame: Day 1
EM includes home infusion guide, infusion diary, and safety and emergency plan.
Percentage of Home Infusion Nurses who Utilize the EM
Time frame: Day 1
EM includes home infusion guide, infusion diary, and safety and emergency plan.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Oct 2026.
- Click here for more information about this trial in easy-to-understand language.Link provided by the sponsor.
- Click here to ask Takeda's chatbot for comprehensive and easy-to-understand information about clinical trials - even across products and indications - in your local language.Link provided by the sponsor.
- Individual participant dataSharing planned: Yes. Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Study Director study director
Takeda
Questions about this trial
What is this clinical trial studying?
An observational study of Gaucher Disease, run by Takeda.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 1 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Takeda Contact (+1-877-825-3327, medinfoUS@takeda.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Participants and Caregivers Aware of Educational Material (EM) (time frame: Day 1). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Oct 2026; AzTrials last refreshed its copy on 7 Nov 2026.