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

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。子项目和研究者（PI）可能从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。所列机构为中心，不一定是研究者所在机构。本临床试验的主要目的是确定与安慰剂相比，艾度硫酸酯酶（DRX 006 A）酶替代疗法治疗II型粘多糖沉积症（MPS II或亨特氏综合征）患者的疗效。艾度硫酸酯酶将以0.5 mg/kg的剂量每周输注给药。本研究的次要目的包括1.以相同的0.5 mg/kg剂量和2.在大型MPS II患者人群中评价艾度硫酸酯酶治疗的2种给药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.