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

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

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

Joseph Muenzer,Muge Gucsavas-Calikoglu,Shawn E McCandless,Thomas J Schuetz,Alan Kimura,

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 week with the 2nd and 3rd groups receiving 0.5 and 1.5 mg/kg, respectively. After 24 weeks the placebo-treated patients were changed to idursulfase at the dose of their group. The primary endpoint was a change from baseline in urinary excretion of glycosaminoglycans. Results were pooled for analysis by ANOVA and compared to baseline. RESULTS: Urinary glycosaminoglycans were reduced within 2 weeks of initiating idursulfase and were decreased 49% after 48 weeks of treatment (P<0.0001). Both liver and spleen volume were decreased at 24 weeks (P<0.01) and 48 weeks (P<0.001). The 6-minute walk test distance increased an average of 48 meters after 48 weeks (P=0.013). Six patients in the higher dose groups developed IgG antibodies that did not influence the clinical effects of idursulfase. CONCLUSIONS: This study describes the first experience with enzyme replacement therapy for the treatment of patients with MPS II. Idursulfase was generally well tolerated and was associated with reductions in urine glycosaminoglycans levels and organ size, as well as an increased 6-minute walk test distance.

A phase I/II clinical trial of enzyme replacement therapy in mucopolysaccharidosis II (Hunter syndrome). Publishing Authors By Initials

J Muenzer,M Gucsavas-Calikoglu,SE McCandless,TJ Schuetz,A Kimura,

For similar tissues: lymphoid tissue: spleen research abstracts see: tissues: lymphoid tissue: spleen research

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

PUBLICATION TYPE: Research Support, Non-U.S. Gov

Journal: Molecular genetics and metabolism

VOLUME: 90

Page Numbers: 329-37

Journal Abbreviation: Mol. Genet. Metab.

ISSN: 1096-7192

DAY: 20

MONTH: 12

YEAR: 2006

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

Number of References:

LANGUAGE: eng

NlmUniqueID: 9805456

A phase I/II clinical trial of enzyme replacement therapy in mucopolysaccharidosis II (Hunter syndrome). Keywords Mesh Terms:

KEYWORDS: Spleen

MESH TERMS: pathology

Chemical & Substance for Abstract: A phase I/II clinical trial of enzyme replacement therapy in mucopolysaccharidosis II (Hunter syndrome). Information

Substance Name: Iduronate Sulfatase

Registry Number: EC 3.1.6.13

Grant and Affiliation Information for A phase I/II clinical trial of enzyme replacement therapy in mucopolysaccharidosis II (Hunter syndrome).

AFFILIATION: University of North Carolina, Department of Pediatrics, CB # 7487, Chapel Hill, NC 27599-7487, USA. muenzer@med.unc.edu <muenzer@med.unc.edu>

Country: United States

AGENCY: United States NCRR

GRANT: RR00046

ACRONYM: RR

MEDLINETA: Mol Genet Metab

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