VPRIV® Non-Interventional Study in Patients Previously Treated With Other Enzyme Replacement Therapies (ERTs)/Substrate Reduction Therapies (SRTs)
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 发起方
- Shire
- 入组人数
- 2
- 试验地点
- 1
- 主要终点
- Number of Participants with Adverse Events (AEs) Following the Transition From Other ERTs/SRTs to VPRIV
研究概览
简要总结
The main aim of this study is to describe the safety profile of velaglucerase alfa (VPRIV) in participants with Gaucher disease type 1. Participants will be transitioning from other enzyme replacement therapies or substrate reduction therapies to VPRIV. Some participants may have already transitioned to treatment with VPRIV before this study started.
In this study, data on VPRIV will be collected from the medical records of participants who already transitioned to VPRIV before this study started. Other participants will join this study when they transition to VPRIV. All participants will be followed to allow for 12 months of observation from time of transition to VPRIV.
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Other
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant with GD1 currently being treated with an ERT/SRT other than VPRIV for at least 6 months before baseline enrolment; or participant previously treated with another ERT/ SRT for at least 6 months prior to transitioning to VPRIV..
- •Participant or legally authorized representative has provided written informed consent.
排除标准
- •In the opinion of the investigator, participant is at high risk of non-compliance.
- •In the opinion of the investigator, participant is unsuitable in any other way to participate in this study.
- •Participant is pregnant.
结局指标
主要结局
Number of Participants with Adverse Events (AEs) Following the Transition From Other ERTs/SRTs to VPRIV
时间窗: Baseline up to 12 months
An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. Number of participants transitioning from other ERTs/SRTs to VPRIV with AEs will be reported.
次要结局
- Change From Baseline in Use of Glucosylspingosine (Lyso-Gb1) Biomarker(Baseline, Month 12)
- Change From Baseline in Gaucher Disease Questionnaire Patient Reported Outcomes (PRO) Score at Month 12(Baseline, Month 12)