Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 50
- 试验地点
- 28
- 主要终点
- Long-term Effectiveness of Vestronidase Alfa
研究概览
简要总结
The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersensitivity reactions and immunogenicity of vestronidase alfa.
详细描述
The Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP) is a global, prospective, multicenter, longitudinal protocol designed to characterize MPS VII disease presentation and progression, assess long-term effectiveness and safety of vestronidase alfa, including hypersensitivity reactions and immunogenicity , as well as prospectively investigate longitudinal change across biomarker(s), clinical assessments, and patient/ caregiver-reported outcome measures in a representative population. The aim of this DMP is to collect data on patients with MPS VII to provide a comprehensive dataset on the clinical presentation, heterogeneity, and disease progression, and meaningful standardized ICH GCP-quality data collected in-clinic across multiple sites globally. The DMP is not a randomized study and both treated and untreated patients will be enrolled.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of MPS VII based on laboratory diagnosis, including either enzymatic or mutation analysis.
- •Willing and able to provide written informed consent or, in the case of patients under the age of 18 (or below adult ages as defined by local laws and regulations) or patients >18 years of age who have cognitive deficiencies, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the DMP has been explained, and prior to any research-related procedures.
- •Willing to comply with DMP visit schedule.
排除标准
- •Concurrent participation in other pharmaceutical company-sponsored interventional clinical trial unless approved by Ultragenyx.
研究组 & 干预措施
Patients with MPS VII receiving vestronidase-alfa
via prescription, or early access/ compassionate use program
干预措施: No Intervention (Other)
Patients with MPS VII not receiving vestronidase-alfa
no treatment or treatment other than vestronidase alfa
干预措施: No Intervention (Other)
结局指标
主要结局
Long-term Effectiveness of Vestronidase Alfa
时间窗: 10 years
To evaluate longitudinal change in biomarker(s), clinical assessments and patient/caregiver reported outcomes to examine the effectiveness of vestronidase alfa
Clinical Course of MPS VII Disease
时间窗: 10 years
To characterize MPS VII disease presentation and progression over time in patients treated and not treated with vestronidase alfa
Long-term Safety of Vestronidase Alfa
时间窗: 10 years
Hypersensitivity reactions, immunogenicity and other safety outcomes will be assessed to examine the long-term safety of vestronidase alfa.
次要结局
未报告次要终点