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NCT Number: NCT05669729

A Survey to Assess Participants', Caregivers', and Nurses' Use and Understanding of Educational Material on Velaglucerase Alfa (VPRIV) Home Infusion

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.

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Key information

Conditions

Gaucher Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Sphingolipidoses

Age range

2 year and older

Sex eligibility

All sexes

Study type

Observational

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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).

Treatment and study plan

Primary outcomes

  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.

Other outcomes

  1. Usefulness of the EM for Participants and Caregivers

    Time frame: Day 1

    EM includes home infusion guide, infusion diary, and safety and emergency plan. Usefulness will be assessed using the 12-item participant and caregiver questionnaire, and 15-item home infusion nurse questionnaire.

  2. Usefulness of the EM for Home Infusion Nurses

    Time frame: Day 1

    EM includes home infusion guide, infusion diary, and safety and emergency plan. Usefulness will be assessed using the 12-item participant and caregiver questionnaire, and 15-item home infusion nurse questionnaire.

Study contacts

Contact information is provided by the study sponsor or research team.

Takeda Contact

CONTACT

[email protected]

+1-877-825-3327

Sponsors and collaborators

Lead sponsor

Takeda

Industry

Registry information

Official study 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

Important dates

Study start
2026
Primary completion
2027
Study completion
2027
First posted
Jan 3, 2023
Registry last updated
Apr 3, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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