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.
Study Type
OBSERVATIONAL
Enrollment
21
Amrita Institute of Medical Sciences & Research Centre (AIMS)
Kochi, Kerala, India
All India Institute of Medical Sciences
New Delhi, India
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).
Time frame: 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.
Time frame: Baseline up to approximately 12 months
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.
Time frame: 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.
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Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: Baseline up to approximately 12 months