Studies
Study First Submitted Date | 2022-12-20 |
Study First Posted Date | 2023-01-03 |
Last Update Posted Date | 2023-06-22 |
Start Month Year | March 4, 2024 |
Primary Completion Month Year | September 5, 2025 |
Verification Month Year | June 2023 |
Verification Date | 2023-06-30 |
Last Update Posted Date | 2023-06-22 |
Detailed Descriptions
Sequence: | 20808217 |
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
Sequence: | 52391683 |
Name | Gaucher Disease |
Downcase Name | gaucher disease |
Id Information
Sequence: | 40315405 |
Id Source | org_study_id |
Id Value | TAK-669-4018 |
Design Groups
Sequence: | 55838489 |
Title | VPRIV® Home Infusion |
Description | 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. |
Keywords
Sequence: | 80168462 |
Name | Drug Therapy |
Downcase Name | drug therapy |
Design Outcomes
Sequence: | 178194016 | Sequence: | 178194017 | Sequence: | 178194018 | Sequence: | 178194019 | Sequence: | 178194020 | Sequence: | 178194021 | Sequence: | 178194022 | Sequence: | 178194023 |
Outcome Type | primary | Outcome Type | primary | Outcome Type | primary | Outcome Type | primary | Outcome Type | primary | Outcome Type | primary | Outcome Type | other | Outcome Type | other |
Measure | Percentage of Participants and Caregivers Aware of Educational Material (EM) | Measure | Percentage of Participants and Caregivers who Understand the EM | Measure | Percentage of Participants and Caregivers who Utilize the EM | Measure | Percentage of Home Infusion Nurses Aware of the EM | Measure | Percentage of Home Infusion Nurses who Understand the EM | Measure | Percentage of Home Infusion Nurses who Utilize the EM | Measure | Usefulness of the EM for Participants and Caregivers | Measure | Usefulness of the EM for Home Infusion Nurses |
Time Frame | Day 1 | Time Frame | Day 1 | Time Frame | Day 1 | Time Frame | Day 1 | Time Frame | Day 1 | Time Frame | Day 1 | Time Frame | Day 1 | Time Frame | Day 1 |
Description | EM includes home infusion guide, infusion diary, and safety and emergency plan. | Description | EM includes home infusion guide, infusion diary, and safety and emergency plan. | Description | EM includes home infusion guide, infusion diary, and safety and emergency plan. | Description | EM includes home infusion guide, infusion diary, and safety and emergency plan. | Description | EM includes home infusion guide, infusion diary, and safety and emergency plan. | Description | EM includes home infusion guide, infusion diary, and safety and emergency plan. | Description | 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. | Description | 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. |
Browse Conditions
Sequence: | 194324371 | Sequence: | 194324372 | Sequence: | 194324373 | Sequence: | 194324374 | Sequence: | 194324375 | Sequence: | 194324376 | Sequence: | 194324377 | Sequence: | 194324378 | Sequence: | 194324379 | Sequence: | 194324380 | Sequence: | 194324381 | Sequence: | 194324382 | Sequence: | 194324383 | Sequence: | 194324384 | Sequence: | 194324385 |
Mesh Term | Gaucher Disease | Mesh Term | Sphingolipidoses | Mesh Term | Lysosomal Storage Diseases, Nervous System | Mesh Term | Brain Diseases, Metabolic, Inborn | Mesh Term | Brain Diseases, Metabolic | Mesh Term | Brain Diseases | Mesh Term | Central Nervous System Diseases | Mesh Term | Nervous System Diseases | Mesh Term | Metabolism, Inborn Errors | Mesh Term | Genetic Diseases, Inborn | Mesh Term | Lipidoses | Mesh Term | Lipid Metabolism, Inborn Errors | Mesh Term | Lysosomal Storage Diseases | Mesh Term | Metabolic Diseases | Mesh Term | Lipid Metabolism Disorders |
Downcase Mesh Term | gaucher disease | Downcase Mesh Term | sphingolipidoses | Downcase Mesh Term | lysosomal storage diseases, nervous system | Downcase Mesh Term | brain diseases, metabolic, inborn | Downcase Mesh Term | brain diseases, metabolic | Downcase Mesh Term | brain diseases | Downcase Mesh Term | central nervous system diseases | Downcase Mesh Term | nervous system diseases | Downcase Mesh Term | metabolism, inborn errors | Downcase Mesh Term | genetic diseases, inborn | Downcase Mesh Term | lipidoses | Downcase Mesh Term | lipid metabolism, inborn errors | Downcase Mesh Term | lysosomal storage diseases | Downcase Mesh Term | metabolic diseases | Downcase Mesh Term | lipid metabolism disorders |
Mesh Type | mesh-list | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor |
Sponsors
Sequence: | 48524726 |
Agency Class | INDUSTRY |
Lead Or Collaborator | lead |
Name | Takeda |
Overall Officials
Sequence: | 29400958 |
Role | Study Director |
Name | Study Director |
Affiliation | Takeda |
Central Contacts
Sequence: | 12065720 |
Contact Type | primary |
Name | Takeda Contact |
Phone | +1-877-825-3327 |
medinfoUS@takeda.com | |
Role | Contact |
Eligibilities
Sequence: | 30892794 |
Sampling Method | Non-Probability Sample |
Gender | All |
Minimum Age | 2 Years |
Maximum Age | N/A |
Healthy Volunteers | No |
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. |
Criteria | 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). |
Adult | True |
Child | True |
Older Adult | True |
Calculated Values
Sequence: | 254135911 |
Registered In Calendar Year | 2022 |
Were Results Reported | False |
Has Single Facility | False |
Minimum Age Num | 2 |
Minimum Age Unit | Years |
Number Of Primary Outcomes To Measure | 6 |
Number Of Other Outcomes To Measure | 2 |
Designs
Sequence: | 30638538 |
Observational Model | Cohort |
Time Perspective | Cross-Sectional |
Links
Sequence: | 4404681 |
Url | https://clinicaltrials.takeda.com/study-detail/e895407fd93e49c7?idFilter=%5B%22TAK-669-4018%22%5D |
Description | To obtain more information on the study, click here/on this link |
Responsible Parties
Sequence: | 29005148 |
Responsible Party Type | Sponsor |
Ipd Information Types
Sequence: | 3351836 | Sequence: | 3351837 | Sequence: | 3351838 | Sequence: | 3351839 |
Name | Study Protocol | Name | Statistical Analysis Plan (SAP) | Name | Informed Consent Form (ICF) | Name | Clinical Study Report (CSR) |