SAFETY AND EFFICACY STUDY OF FABRAZYMER IN PATIENTS WITH FABRY DISEASE

FABRAZYMER 在法布瑞病患者中的安全性和有效性研究

• 批准号: 7379342
• 负责人: C RONALD SCOTT
• 金额: $ 0.58万
• 依托单位: UNIVERSITY OF WASHINGTON
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-04-01 至 2007-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7379342
• 关键词: SAFETY; EFFICACY; STUDY; FABRAZYMER; PATIENTS

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Fabry disease is a lysosomal storage disease that involves severe kidney disease, cardiac disease, cerebrovascular disease, and early death for affected individuals. This study is a multicenter, multinational, open-label trial to confirm the safety and effectiveness of Fabrazyme in patients with advanced Fabry disease who received either Fabrazyme or placebo as treatment for Fabry disease in the AGAL-008-00 trial. The primary objective is to evaluate the stabilization of renal function with Fabrazyme.

该子项目是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。子弹和调查员（PI）可能已经从其他NIH来源获得了主要资金，因此可以在其他清晰的条目中代表。列出的机构适用于该中心，这不一定是调查员的机构。 Fabry病是一种溶酶体储存疾病，涉及严重的肾脏疾病，心脏病，脑血管疾病和患者的早期死亡。这项研究是一项多中心，跨国标签的试验，旨在确认在AGAL-008-00试验中接受Fabrazyme或安慰剂治疗的晚期Fabry疾病患者的Fabrazyme的安全性和有效性。 主要目的是评估使用Fabrazyme的肾功能稳定。