Gaucher Disease Outcome Survey (GOS)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- Shire
- 入组人数
- 1,257
- 试验地点
- 2
- 主要终点
- Increase in Bone Mineral Density (BMD)
研究概览
简要总结
The Gaucher Outcomes Survey (GOS) is an ongoing observational, international, multi-center, long-term Registry of Patients with Gaucher Disease irrespective of their treatment status or type of treatment received. No experimental intervention is involved. Patients undergo clinical assessments and receive care as determined by the patients' treating physician.
The objectives of the registry include to evaluate the safety and long-term effectiveness of velaglucerase alfa, to characterize patients receiving velaglucerase alfa or other Gaucher Disease-specific treatments, to gain a better understanding of the natural history of GD and to serve as a database for evidence-based management of Gaucher Disease over time in real-life clinical practice.
详细描述
20 MAY 2020: The temporary enrollment stop of new patients into this study due to the COVID-10 pandemic has been lifted in one or more countries/sites, and the study is now again enrolling new patients. However, some countries/sites may still have paused the enrollment of new patients due to the pandemic.
24 APRIL 2020: Enrollment of new patients into this study has been paused due to the COVID-19 situation. The duration of this pause is dependent on the leveling and control of the COVID-19 pandemic.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients of any age or gender with confirmed diagnosis (biochemical and/or genetic) of Gaucher disease
- •Signed and dated written informed consent from the patient or, for patients aged <18 years (<16 years in the United Kingdom [UK]), their parent and/or legally authorized representatives (LAR), and assent of the minor where applicable. Legally authorized representatives are also applicable for cognitively impaired patients.
排除标准
- •- Patients currently enrolled in ongoing blinded clinical trials (drugs or devices; includes all blinded trials)
研究组 & 干预措施
GOS Participants
GOS is a disease specific registry open to all Gaucher patients irrespective of treatment status or type of treatment
结局指标
主要结局
Increase in Bone Mineral Density (BMD)
时间窗: Baseline to one year for up to 20 years
Bone mineral density will be assessed.
Decrease in Liver Volume
时间窗: Baseline to one year for up to 20 years
Liver volume will be assessed by abdominal imaging.
Number of Participants With Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: Baseline to one year for up to 20 years
Treatment-emergent adverse events (TEAEs) are defined as adverse events (AEs) that either commenced or worsened following the first dose of VPRIV.
Increase of Platelet Count
时间窗: Baseline to one year for up to 20 years
Platelet count will be assessed.
Decrease in Spleen Volume
时间窗: Baseline to one year for up to 20 years
Spleen volume will be assessed by abdominal imaging.
Number of Participants With Infusion-related Reactions (IRRs)
时间窗: Baseline to one year for up to 20 years
An IRR is defined as an AE that has been assessed as at least possibly related to treatment with VPRIV and occurs during an infusion or up to 24 hours post-VPRIV infusion.
Increase of Hemoglobin Concentration
时间窗: Baseline to one year for up to 20 years
Hemoglobin concentration will be assessed.
次要结局
未报告次要终点