Phase 1 Open Label Study in Ten Mps I Patients 12711 Study Objectives and Design

The first study of Aldurazyme was designed as an open-label study of weekly intravenous infusions of rhIDU at a dose of 0.58 mg/kg in 6 to 10 patients of age 5 years or greater and representing a wide range of disease severity [12]. Given the open-label design, only objectively measured clinical endpoints were proposed, and the analysis was based on comparing pretreatment with posttreatment measurements for the various endpoints. The primary endpoint variables were quantitative measures of storage, including liver or spleen size and urinary GAG excretion. Liver or spleen size is enlarged in MPS I due to storage, and a reduction in organ size was measured by MRI. Urinary GAG excretion is elevated in MPS I patients, and a reduction in urinary GAG excretion would represent a reduction in renal storage. Secondary endpoint variables included sleep apnea, shoulder, knee, and elbow maximum range of motion, cardiac evaluations (a scoring system of history, physical, echocardiography findings, and the New York Heart Association

[NYHA] classification), and eye disease. In prepubertal patients, height and weight growth velocity were also studied. Safety evaluations included the standard clinical laboratory studies, adverse event monitoring, assessments for antibodies to rhIDU, and complement activation.

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