IDURONATE-2-SULFATASE ENZYME REPLACEMENT THERAPY IN PATIENTS WITH MPS II

这个子项目是利用由NIH/NCRR资助的中心拨款提供的资源的许多研究子项目之一。子项目和调查员(PI)可能从另一个NIH来源获得了主要资金，因此可能会出现在其他CRISE条目中。列出的机构是针对中心的，而不一定是针对调查员的机构。这项临床试验的主要目标是确定与安慰剂相比，使用艾杜硫酶(DRX006A)的酶替代疗法治疗II型粘多糖病(MPS II或Hunter综合征)的疗效。艾度硫酶每周一次输注，剂量为0.5毫克/公斤。这项研究的次要目标包括：1.在相同的0.5 mg/kg剂量下，每隔一周给药一次的艾杜硫酶方案的疗效的测定；2.在MPS II患者群体中评估艾杜硫酶两种剂量方案的安全性和耐受性。

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. The primary objective of this clinical trial is to determine the efficacy of enzyme replacement therapy with idursulfase (DRX006A) in the treatment of patients with Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome) as compared to placebo. Idursulfase will be administered as a weekly infusion at a dose of 0.5 mg/kg. The secondary objectives of the study include 1. The determination of the efficacy of an eveyr other week regimen of idursulfase given at the same 0.5 mg/kg dose and 2. The evaluation of the safety and tolerability of the 2 dosing regimens of idursulfase therapy in a large MPS II patient population.