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| ID | Type | Description | Link |
|---|---|---|---|
| EUPAS42338 | Registry Identifier | EUPAS |
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The decision was taken after careful consideration relating to recruitment challenges that could impact the scientific value of the study.
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The main aim of this study is to describe the safety profile of velaglucerase alfa (VPRIV) in participants with Gaucher disease type 1. Participants will be transitioning from other enzyme replacement therapies or substrate reduction therapies to VPRIV. Some participants may have already transitioned to treatment with VPRIV before this study started.
In this study, data on VPRIV will be collected from the medical records of participants who already transitioned to VPRIV before this study started. Other participants will join this study when they transition to VPRIV. All participants will be followed to allow for 12 months of observation from time of transition to VPRIV.
The study sponsor will not be involved in how participants are treated but will provide instructions on how the clinics will record what happens during the study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| VPRIV Participants | Participants who has been transitioned from ERTs/SRTs to VPRIV, the data will be collected retrospectively from time of transition until the point at which participant begins in this study and then will be followed up prospectively for 12 months. |
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| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants with Adverse Events (AEs) Following the Transition From Other ERTs/SRTs to VPRIV | An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. Number of participants transitioning from other ERTs/SRTs to VPRIV with AEs will be reported. | Baseline up to 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Use of Glucosylspingosine (Lyso-Gb1) Biomarker | Change in use of Lyso-Gb1 by participants following the transition from other ERTs/SRTs to velaglucerase alfa (VPRIV) will be assessed. | Baseline, Month 12 |
| Change From Baseline in Gaucher Disease Questionnaire Patient Reported Outcomes (PRO) Score at Month 12 |
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Inclusion Criteria:
Exclusion Criteria:
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Participants included in the study will have a diagnosis of Type 1 GD and be in enzyme/substrate replacement therapy other than VPRIV, for at least 6 months prior to enrolment in the study, or have been previously treated with an ERT/SRT other than VPRIV for a minimum of 6 months prior to transitioning to VPRIV.
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Shire | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alberta/Medical Genetics Clinic | Edmonton | Alberta | T6G 2H7 | Canada |
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| Label | URL |
|---|---|
| To obtain more information on the study, click here/on this link | View source |
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De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be re-identified (due to the limited number of study participants).
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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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Gaucher disease questionnaire (PRO) scores will be calculated for each adult participant. The questionnaire asks how much the participant is affected by the Gaucher disease. A total summated score is calculated and ranges from 0 to 100 with higher scores indicating more severe impairment and worse quality of life. |
| Baseline, Month 12 |
| 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 |