A phase I/II clinical trial of enzyme replacement therapy in mucopolysaccharidosis II (Hunter syndrome)

J Muenzer, M Gucsavas-Calikoglu… - Molecular genetics and …, 2007 - Elsevier
OBJECTIVE: To evaluate the safety and explore the efficacy of idursulfase (recombinant
human iduronate-2-sulfatase) treatment for mucopolysaccharidosis II (MPS II). STUDY
DESIGN: Twelve patients were enrolled into a randomized, double-blind, placebo-controlled
trial for 24 weeks followed by an open-label extension study. Three groups of 4 patients
were enrolled sequentially, with 3 patients in each group receiving idursulfase and 1 patient
receiving placebo. The first group received idursulfase at 0.15 mg/kg infused every other …