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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
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| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants and Caregivers Aware of Educational Material (EM) | EM includes home infusion guide, infusion diary, and safety and emergency plan. | Day 1 |
| Percentage of Participants and Caregivers who Understand the EM | EM includes home infusion guide, infusion diary, and safety and emergency plan. | Day 1 |
| Percentage of Participants and Caregivers who Utilize the EM | EM includes home infusion guide, infusion diary, and safety and emergency plan. | Day 1 |
| Percentage of Home Infusion Nurses Aware of the EM | EM includes home infusion guide, infusion diary, and safety and emergency plan. | Day 1 |
| Percentage of Home Infusion Nurses who Understand the EM | EM includes home infusion guide, infusion diary, and safety and emergency plan. | Day 1 |
| Percentage of Home Infusion Nurses who Utilize the EM | EM includes home infusion guide, infusion diary, and safety and emergency plan. | Day 1 |
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| Measure | Description | Time Frame |
|---|---|---|
| Usefulness of the EM for Participants and Caregivers | 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. | Day 1 |
| Usefulness of the EM for Home Infusion Nurses |
Inclusion criteria:
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).
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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.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Takeda Contact | Contact | +1-877-825-3327 | medinfoUS@takeda.com |
| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
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| Label | URL |
|---|---|
| Click here for more information about this trial in easy-to-understand language. | View source |
| 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. | View source |
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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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IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/ For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
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| ID | Term |
|---|---|
| D005776 | Gaucher Disease |
| ID | Term |
|---|---|
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
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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. |
| Day 1 |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D052439 | Lipid Metabolism Disorders |