A multicenter, open-label study evaluating safety and clinical outcomes in children (1.4-7.5 years) with Hunter syndrome receiving idursulfase enzyme replacement therapy.

ABSTRACT:

Purpose

The primary objective of this study was to determine the safety of idursulfase in Hunter syndrome patients aged 5 years or younger.

Methods

Idursulfase (0.5 mg/kg) was administered intravenously on a weekly basis (52 infusions per patient) in an open-label study. Safety monitoring included adverse events, anti-idursulfase antibodies, vital signs, physical examination, 12-lead electrocardiogram, concomitant medications or procedures, and laboratory testing (clinical chemistry, hematology, and urinalysis). The following exploratory efficacy outcomes were assessed at baseline and at weeks 18 or 36 or 53: urinary glycosaminoglycan levels, liver or spleen size, developmental milestones, and growth indices. Pharmacokinetic parameters were assessed at week 27.

Results

SUBMITTER: Giugliani R 

PROVIDER: S-EPMC4052427 | biostudies-literature | 2014 Jun

REPOSITORIES: biostudies-literature