An Open-Label Treatment Protocol With UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy for an Advanced Stage Mucopolysaccharidosis Type 7 (MPS 7) Patient

Joyce Fox1 个研究点分布在 1 个国家开始时间: 2014年3月27日最近更新: 2017年2月6日

适应症

试验速览

阶段: 不适用
状态: No Longer Available
发起方
试验地点: 1

研究概览入排标准研究者研究点记录与标识符相似试验

研究概览

简要总结

Emergency access granted to treat a single patient with advanced Mucopolysaccharidosis Type 7 with UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy via IV administration every other week (QOW) for up to 144 weeks.

研究设计

研究类型: Expanded Access

入排标准

性别: Male
接受健康志愿者: 否

入选标准

•1 specific patient
•Confirmed diagnosis of MPS 7 based on leukocyte or fibroblast glucuronidase enzyme assay or genetic testing confirming diagnosis.
•Written informed consent of parent(s) or legal guardian(s)

排除标准

未提供

研究者

发起方

Joyce Fox

申办方类型

Other

责任方

Sponsor Investigator

主要研究者

Joyce Fox

Chief of Medical Genetics at Cohen Children's Medical Center

Northwell Health

研究点 (1)

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