NL-OMON36408已完成不适用
A Multicenter, Multinational, Longitudinal Clinical Assessment Study of Subjects with Mucopolysaccharidosis IVA (Morquio Syndrome). - A Cross-sectional Clinical Assessment of Subjects with MPS IV A.
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- BioMarin
- 入组人数
- 25
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Observational
入排标准
- 年龄范围
- 2 至 99（—）
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
- •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
- •Previous hematopoietic stem cell transplant (HSCT).
- •Concurrent disease or condition that would interfere with study participation or pose a safety
研究者
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