Survey Study for Velaglucerase Alfa (VPRIV) in Japan

Study Overview
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Brief Summary
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The objective of this post-marketing survey study is to collect data to determine the safety and efficacy of velaglucerase alfa (VPRIV) in participants with Gaucher disease who are new to therapy or have been switched from another therapeutic agent for Gaucher disease.

Official Title
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VPRIV Drug Use-Result Survey (Japan)

Conditions
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Gaucher Disease

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male or female participants with a confirmed diagnosis of Gaucher disease Participants who are either naïve to treatment or participants that have been treated with another therapeutic agent for Gaucher disease Participants who start VPRIV treatment or transition from VPRIV clinical studies during the enrollment period Exclusion Criteria: -
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 |
| --- | --- |
| Gaucher Disease Participants Treated With VPRIV<br>Participants with Gaucher disease will be enrolled in this survey, who are in VPRIV treatment-naïve therapy or have been switched from another therapeutic agent for Gaucher disease. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAE) Following Initiation of Treatment With Velaglucerase Alfa (VPRIV) | An AE is any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product.An SAE is any event that results in: death; life-threatening; requires inpatient hospitalisation or results in prolongation of existing hospitalisation; persistent or significant disability/incapacity; a congenital anomaly/birth defect or a medically important event. | Baseline up to end of the study (8 years) | 
| Number of Participants With Absence/presence of Anti-velaglucerase Alfa Antibodies | Effect of anti-velaglucerase alfa antibodies (including IgGs) will be performed at the physician's discretion per standard clinical practice. Immunoglobulin E (IgE) isotype-specific antibodies will also be measured when clinically indicated (for example, adverse event, serious adverse event or possible of lack of efficacy). | Baseline up to end of the study (8 years) | 
| Number of Participants With Clinically Significant Change in Laboratory Assessment | Clinically significant changes in laboratory assessments will be reported. | Baseline up to end of the study (8 years) | 
| Number of Participants With Hypersensitivity Reactions | Hypersensitivity reactions are defined as events of drug allergy, angioedema, anaphylactic reaction, anaphylactic shock, anaphylactoid reaction, anaphylaxis prophylaxis, anaphylaxis treatment, drug reaction with eosinophilia and systemic symptoms. | Baseline up to end of the study (8 years) | 
| Occurrences of Pregnancies/breastfeeding for Women of Child-bearing Potential | Pregnancy testing for women of child-bearing potential will be collected throughout the survey. | Baseline up to end of the study (8 years) | 
| Change From Baseline in Hemoglobin Concentration | Hemoglobin concentration will be assessed. | Baseline, Every 12 weeks up to 8 years | 
| Change From Baseline in Platelet Count | Platelet count will be assessed. | Baseline, Every 12 weeks up to 8 years | 
| Change From Baseline in Liver Volume | Liver volume will be measured at the physician's discretion per standard clinical practice. | Baseline, Every 24 weeks up to 8 years | 
| Change From Baseline in Spleen Volume | Spleen volume will be measured at the physician's discretion per standard clinical practice. | Baseline, Every 24 weeks up to 8 years | 
| Change From Baseline in Bone Density | Bone density will be assessed at the physician's discretion per standard clinical practice. | Baseline, Every 52 weeks up to 8 years | 
| Number of Participants With Infusion-related Reactions (IRRs) | Infusion-related reactions are defined as reactions occurring up to 24 hours after the start of the infusion. | Baseline up to end of the study (8 years) | 
| Number of Participants with anti-velaglucerase alfa antibodies | Anti-velaglucerase alfa antibodies will be assessed. | Baseline up to end of study (8 years) |