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

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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.