NCT00787995终止不适用
A Multicenter, Multinational, Longitudinal Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
适应症
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 353
- 主要终点
- Respiratory Function
研究概览
简要总结
This multicenter, multinational, longitudinal study will quantify endurance and respiratory function in subjects diagnosed with MPS IVA and will better characterize the spectrum of symptoms and biochemical abnormalities in MPS IVA disease over time.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Willing and able to provide written, signed informed consent, or in the case of subjects age < 18 years, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.
- •Documented history of reduced GALNS activity relative to the normal range of the laboratory performing the assay, or documented result of molecular genetic testing confirming diagnosis of MPS IVA.
- •Willing and able to comply with all study procedures.
排除标准
- •Use of any investigational product or investigational medical device within 30 days prior to screening.
- •Previous hematopoietic stem cell transplant (HSCT).
- •Concurrent disease or condition that would interfere with study participation or pose a safety concern.
结局指标
主要结局
Respiratory Function
时间窗: Study Visit
Endurance
时间窗: Study Visit
次要结局
未报告次要终点
研究者
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