NCT01356537已完成不适用
Non Interventional Study of VPRIV® (Velaglucerase Alfa) Home Therapy in Patients With Gaucher's Disease
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Shire
- 入组人数
- 34
- 试验地点
- 11
- 主要终点
- Patient satisfaction estimated on a 10-ary Likert scale, quality of life estimated by SF-36 questionnaire
研究概览
简要总结
The purpose of this study is to proof increasing patient satisfaction and preservation of quality of life in patients with Gaucher's Disease receiving their enzyme replacement therapy with VPRIV (Velaglucerase alfa)at their home setting compared to receiving the infusions at the clinic or at doctor's practice.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female patients with a confirmed diagnosis of Gaucher disease type 1
- •Age> 2 years
- •patients who have at least 3 infusions (6 weeks) at least 5-year or 5-6 infusions (10-12 weeks) at 2 - to 4-year patients have received VPRIV ® and tolerate well
- •The patient is compliant, the previous VPRIV ® infusions were / performed approximately every 2 weeks in the center during office visits
- •The patient was already before inclusion in this study for a home infusion therapy and has consented to (or their legal representative)
- •The patient / be lawful. Representative has consented in writing to participate in this study.
- •Exclusion criteria:
- •The patient is participating in a clinical trial with a medicinal product
排除标准
- 未提供
结局指标
主要结局
Patient satisfaction estimated on a 10-ary Likert scale, quality of life estimated by SF-36 questionnaire
时间窗: comparison of baseline to 12 months value
次要结局
- Number (per infusion) and severity of infusion-related side effects(baseline compared to 12 months)
研究者
研究点 (11)
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