Post Marketing Surveillance (PMS) Study for Velaglucerase Alfa (VPRIV) in India

Study Overview
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Brief Summary
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The main aim of this study is to measure the safety and to find out the effects of VPRIV in participants with Gaucher disease using both retrospective and prospective data when used in the post-marketing setting and to collect genetic mutation data from participants with Gaucher disease. 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. When the participants start the study, they will visit the study clinic close to approximately 12 months.

Official Title
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A Post Marketing Surveillance (PMS) Study for VPRIV (Velaglucerase Alfa) in India

Conditions
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Gaucher Disease

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Participants with type 1 Gaucher disease prescribed VPRIV according to the investigator's judgment and current Indian Prescribing information (PI) are eligible for this study. Participants or legally authorized representative must provide written informed consent to participate. Exclusion Criteria: - Participants will be excluded from this study if the participant met any of the contraindications included in the current Indian PI for VPRIV.
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 |
| --- | --- |
| VPRIV: All Participants<br>Participants with type 1 Gaucher disease who as part of standard or routine clinical practice, have already received VPRIV retrospectively and will further receive VPRIV therapy according to the investigator's judgment for approximately 12 months will be observed prospectively in this PMS study. | |

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) | An AE is defined as any untoward medical occurrence (including a symptom or disease or an abnormal laboratory finding) in a participant or clinical investigation participants administered a medicinal product and which does not necessarily have a causal relationship with the treatment. A serious adverse event (SAE) is any event that results in: death; life-threatening event; requires inpatient hospitalization or results in prolongation of existing hospitalization; persistent or significant disability/incapacity; results in a congenital anomaly/birth defect or a medically important event. AEs include serious adverse events, unexpected AEs, non-serious adverse events (AEs) will be reported using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on charitable access program (CAP). | Baseline up to approximately 12 months | 
| Number of Participants With Adverse Drug Reactions (ADRs) | An ADR is an AE for which there is at least a reasonable suspicion of a causal relationship between an AE and a suspected medicinal product. Number of participants with ADRs will be reported using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on CAP. | Baseline up to approximately 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline in Hemoglobin (Hb) Concentration Using Both Retrospective and Prospective Data | Hemoglobin concentration will be assessed in participants with Gaucher disease receiving VPRIV using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on CAP. | Baseline up to approximately 12 months | 
| Change From Baseline in Platelet Count Using Both Retrospective and Prospective Data | Platelet count will be assessed in participants with Gaucher disease receiving VPRIV using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on CAP. | Baseline up to approximately 12 months | 
| Change From Baseline in Spleen Size Using Both Retrospective and Prospective Data | Spleen size will be assessed using ultrasound or Magnetic Resonance Image (MRI) in participants with Gaucher disease receiving VPRIV using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on CAP. | Baseline up to approximately 12 months | 
| Change From Baseline in Liver Size Using Both Retrospective and Prospective Data | Liver size will be assessed using ultrasound or MRI in participants with Gaucher disease receiving VPRIV using both retrospective and prospective data when used as standard clinical practice. Baseline is first time participants were dosed on CAP. | Baseline up to approximately 12 months | 
| Treatment History Based on Previous VPRIV Treatment | Presence of treatment history will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP. | At Baseline | 
| Change From Baseline in Hemoglobin (Hb) Concentration Based on Previous VPRIV Treatment | Hemoglobin concentration will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP. | Baseline up to approximately 12 months | 
| Change From Baseline in Platelet Count Based on Previous VPRIV Treatment | Platelet count data will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP. | Baseline up to approximately 12 months | 
| Change From Baseline in Spleen Size Based on Previous VPRIV Treatment | Spleen size date will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP. | Baseline up to approximately 12 months | 
| Change From Baseline in Liver Size Based on Previous VPRIV Treatment | Liver size data will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP. | Baseline up to approximately 12 months | 
| Number of Participants With AEs and SAEs Based on Previous VPRIV Treatment | An AE is defined as any untoward medical occurrence (including a symptom or disease or an abnormal laboratory finding) in a participant or clinical investigation participants administered a medicinal product and which does not necessarily have a causal relationship with the treatment. A SAE is any event that results in: death; life-threatening event; requires inpatient hospitalization or results in prolongation of existing hospitalization; persistent or significant disability/incapacity; results in a congenital anomaly/birth defect or a medically important event. AEs include serious adverse events, unexpected AEs, non-serious AEs will be collected using retrospective data of the previous VPRIV treatment. Baseline is defined as measurement from first time participants was dosed on CAP. Data will be assessed retrospectively based on participant records. | Baseline up to approximately 12 months |