A Post Marketing Surveillance (PMS) Study for VPRIV (Velaglucerase Alfa) in India
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Shire
- 入组人数
- 21
- 试验地点
- 2
- 主要终点
- Number of Participants With Adverse Events (AEs)
研究概览
简要总结
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •Participants will be excluded from this study if the participant met any of the contraindications included in the current Indian PI for VPRIV.
结局指标
主要结局
Number of Participants With Adverse Events (AEs)
时间窗: 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)
时间窗: 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.
次要结局
- Change From Baseline in Spleen Size Using Both Retrospective and Prospective Data(Baseline up to approximately 12 months)
- Change From Baseline in Liver Size Using Both Retrospective and Prospective Data(Baseline up to approximately 12 months)
- Change From Baseline in Platelet Count Based on Previous VPRIV Treatment(Baseline up to approximately 12 months)
- Change From Baseline in Hemoglobin (Hb) Concentration Using Both Retrospective and Prospective Data(Baseline up to approximately 12 months)
- Change From Baseline in Hemoglobin (Hb) Concentration Based on Previous VPRIV Treatment(Baseline up to approximately 12 months)
- Change From Baseline in Platelet Count Using Both Retrospective and Prospective Data(Baseline up to approximately 12 months)
- Change From Baseline in Spleen Size Based on Previous VPRIV Treatment(Baseline up to approximately 12 months)
- Change From Baseline in Liver Size Based on Previous VPRIV Treatment(Baseline up to approximately 12 months)
- Number of Participants With AEs and SAEs Based on Previous VPRIV Treatment(Baseline up to approximately 12 months)
- Treatment History Based on Previous VPRIV Treatment(At Baseline)