A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease

Study Overview
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Brief Summary
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The main aim of this study is to learn if velaglucerase alfa (VPRIV) improves growth and symptoms in participants up to 5 years of age with Gaucher disease. Symptoms will be checked with blood tests. This study is about collecting data available in the participant's medical record as well as data from each participant's ongoing treatment. No study medicines will be provided to participants in this study. 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. When the participants start the study, they will visit the study clinic every 6 months after their first visit.

Official Title
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Gaucher Disease During Infancy and Early Childhood and Experience With Enzyme Replacement Therapy (ERT) Using Velaglucerase Alfa (VPRIV): A Combined Retrospective and Prospective Cohort Study

Conditions
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Gaucher Disease

Intervention / Treatment
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* Other: Standard of Care


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: The participant's caregiver is able and willing to provide informed consent. The participant is male or female younger than or equal to 4 years of age at treatment initiation. The participant has received and confirmed a current diagnosis of GD type 1 or type 3 (biochemically and/or genetically). The participant has been receiving intravenous (IV) Velaglucerase alfa treatment for GD. In the opinion of the investigator, the participant's caregiver is capable of understanding and complying with protocol requirements. The participant's legally acceptable representative signs and dates a written, informed consent form and any required privacy authorization prior to the initiation of any study procedures. Exclusion Criteria: The participant is an immediate family member, study site employee, or is in a dependent relationship with a study site employee who is involved in conduct of this study (e.g., child, sibling) or may consent under duress. The participant is judged by the investigator as being ineligible for any other reason.

Ages Eligible for Study
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Maximum Age: 5 Years

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 |
| --- | --- |
| Standard of Care (SoC)<br>Neonatal and pediatric participants who has been on ERT (VPRIV) will be followed up for 36 months from the time of treatment initiation as per SOC. | Other: Standard of Care<br>* Neonatal and pediatric participants who has been on ERT (VPRIV) will be assessed as per SOC.<br>* Other names: SOC;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline in Hemoglobin (Hb) Level | Increase hemoglobin levels up to 11.0 gram per deciliter (g/dL) will be assessed. | From start of ERT initiation up to 5 years of age | 
| Percent Change From Baseline in Platelet Count Increase | Percent change from baseline for platelet count increase will be assessed. | From start of ERT initiation up to 5 years of age | 
| Percent Change From Baseline in Liver Volume | Percent change from baseline in liver volume will be assessed. | From start of ERT initiation up to 5 years of age | 
| Percent Change From Baseline for Spleen Volume | Percent change from baseline for spleen volume will be assessed. | From start of ERT initiation up to 5 years of age | 
| Percentage of Participants With Growth Normalization | Percentage of participants with growth normalization will be assessed. | From start of ERT initiation up to 5 years of age | 
| Percentage of Participants With Improvement in Bone Disease | Percentage of participants with improvement in bone disease will be assessed. | From start of ERT initiation up to 5 years of age | 
| Percentage of Participants With Improvement in Thrombocytopenia | Percentage of participants with improvement in thrombocytopenia will be assessed. | From start of ERT initiation up to 5 years of age | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | An adverse event (AE) is any untoward medical occurrence in a participant administered a medicinal product and which does not necessarily have to have a causal relationship with this treatment. An SAE is any event that results in: death; life-threatening; requires inpatient hospitalization or results in prolongation of existing hospitalization; persistent or significant disability/incapacity; a congenital anomaly/birth defect or a medically important event. AEs include SAEs, non-serious AEs. | From start of ERT initiation up to 5 years of age |