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The purpose of this study is to proof increasing patient satisfaction and preservation of quality of life in patients with Gaucher's Disease receiving their enzyme replacement therapy with VPRIV (Velaglucerase alfa)at their home setting compared to receiving the infusions at the clinic or at doctor's practice.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Gaucher's Disease under VPRIV |
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| Measure | Description | Time Frame |
|---|---|---|
| Patient satisfaction estimated on a 10-ary Likert scale, quality of life estimated by SF-36 questionnaire | comparison of baseline to 12 months value |
| Measure | Description | Time Frame |
|---|---|---|
| Number (per infusion) and severity of infusion-related side effects | baseline compared to 12 months |
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Inclusion Criteria:
Exclusion criteria:
• The patient is participating in a clinical trial with a medicinal product
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Patients with proven Gaucher's Disease foreseen for home treatment with VPRIV (Velaglucerase alfa) at German Gaucher centers
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Landeskrankenhaus Bregenz | Bregenz | A-6900 | Austria | |||
| Paracelsus Medizinische Privatuniversität Salzburg |
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| Label | URL |
|---|---|
| To obtain more information on the study, click here/on this link. | 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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| Salzburg |
| A-5020 |
| Austria |
| AKH, Allgemeines Krankenhaus der Stadt Wien | Vienna | A-1090 | Austria |
| Medical University of Vienna | Vienna | A-1090 | Austria |
| Medizinische Universität Wien | Vienna | A-1090 | Austria |
| Gemeinschaftspraxis für Hämatologie und Onkologie | Cologne | 51103 | Germany |
| Universitätsklinikum Mainz | Mainz | 55131 | Germany |
| Klinikum rechts der Isar | München | D-81675 | Germany |
| Albrecht-Kossel-Institut für Neuroregeneration (AKos) | Rostock | D-18147 | Germany |
| Helios Klinikum Schwerin | Schwerin | D-19055 | Germany |
| Universitätsklinikum Ulm | Ulm | D-89075 | Germany |
| 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 |