Gaucher Disease
Conditions
Brief summary
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.
Interventions
None listed
Sponsors
Shire
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Adverse Events (AEs) | Baseline up to approximately 12 months | 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). |
| Number of Participants With Adverse Drug Reactions (ADRs) | Baseline up to approximately 12 months | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Spleen Size Using Both Retrospective and Prospective Data | Baseline up to approximately 12 months | 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. |
| Treatment History Based on Previous VPRIV Treatment | At Baseline | Presence of treatment history will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP. |
| Change From Baseline in Liver Size Using Both Retrospective and Prospective Data | Baseline up to approximately 12 months | 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. |
| Change From Baseline in Hemoglobin (Hb) Concentration Based on Previous VPRIV Treatment | Baseline up to approximately 12 months | Hemoglobin concentration will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP. |
| Change From Baseline in Hemoglobin (Hb) Concentration Using Both Retrospective and Prospective Data | Baseline up to approximately 12 months | 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. |
| Change From Baseline in Spleen Size Based on Previous VPRIV Treatment | Baseline up to approximately 12 months | Spleen size date will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP. |
| Change From Baseline in Liver Size Based on Previous VPRIV Treatment | Baseline up to approximately 12 months | Liver size data will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP. |
| Number of Participants With AEs and SAEs Based on Previous VPRIV Treatment | Baseline up to approximately 12 months | 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. |
| Change From Baseline in Platelet Count Based on Previous VPRIV Treatment | Baseline up to approximately 12 months | Platelet count data will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP. |
| Change From Baseline in Platelet Count Using Both Retrospective and Prospective Data | Baseline up to approximately 12 months | 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. |
Countries
India
Outcome results
None listed