Gaucher Disease Outcome Survey (GOS)

Study Overview
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Brief Summary
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The Gaucher Outcomes Survey (GOS) is an ongoing observational, international, multi-center, long-term Registry of Patients with Gaucher Disease irrespective of their treatment status or type of treatment received. No experimental intervention is involved. Patients undergo clinical assessments and receive care as determined by the patients' treating physician. The objectives of the registry include to evaluate the safety and long-term effectiveness of velaglucerase alfa, to characterize patients receiving velaglucerase alfa or other Gaucher Disease-specific treatments, to gain a better understanding of the natural history of GD and to serve as a database for evidence-based management of Gaucher Disease over time in real-life clinical practice.

Detailed Description
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20 MAY 2020: The temporary enrollment stop of new patients into this study due to the COVID-10 pandemic has been lifted in one or more countries/sites, and the study is now again enrolling new patients. However, some countries/sites may still have paused the enrollment of new patients due to the pandemic. 24 APRIL 2020: Enrollment of new patients into this study has been paused due to the COVID-19 situation. The duration of this pause is dependent on the leveling and control of the COVID-19 pandemic.

Official Title
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Gaucher Disease Outcome Survey (GOS)

Conditions
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Gaucher Disease

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients of any age or gender with confirmed diagnosis (biochemical and/or genetic) of Gaucher disease Signed and dated written informed consent from the patient or, for patients aged <18 years (<16 years in the United Kingdom [UK]), their parent and/or legally authorized representatives (LAR), and assent of the minor where applicable. Legally authorized representatives are also applicable for cognitively impaired patients. Exclusion Criteria: - Patients currently enrolled in ongoing blinded clinical trials (drugs or devices; includes all blinded trials)
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| GOS Participants<br>GOS is a disease specific registry open to all Gaucher patients irrespective of treatment status or type of treatment | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Treatment-emergent adverse events (TEAEs) are defined as adverse events (AEs) that either commenced or worsened following the first dose of VPRIV. | Baseline to one year for up to 20 years | 
| Number of Participants With Infusion-related Reactions (IRRs) | An IRR is defined as an AE that has been assessed as at least possibly related to treatment with VPRIV and occurs during an infusion or up to 24 hours post-VPRIV infusion. | Baseline to one year for up to 20 years | 
| Increase of Hemoglobin Concentration | Hemoglobin concentration will be assessed. | Baseline to one year for up to 20 years | 
| Increase of Platelet Count | Platelet count will be assessed. | Baseline to one year for up to 20 years | 
| Decrease in Liver Volume | Liver volume will be assessed by abdominal imaging. | Baseline to one year for up to 20 years | 
| Decrease in Spleen Volume | Spleen volume will be assessed by abdominal imaging. | Baseline to one year for up to 20 years | 
| Increase in Bone Mineral Density (BMD) | Bone mineral density will be assessed. | Baseline to one year for up to 20 years |