NCT00069641

Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)

A Phase II/III, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Weekly and Every Other Week Dosing Regimens of Iduronate-2-Sulfatase Enzyme Replacement Therapy in Patients With MPS II

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 96 (actual)
• Sponsor: Shire · Industry
• Sex: Male
• Age: 5 Years – 25 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to determine whether the administration of iduronate-2-sulfatase enzyme in a weekly or every other week therapy frequency is safe and efficacious in patients with MPS II.

Detailed description

MPS II is a rare, X-linked, lysosomal storage disorder caused by a deficiency in the enzyme iduronate-2-sulfatase. Because of this deficiency, glycosaminoglycans (GAG) accumulate in multiple tissues and organs, resulting in progressive cellular and organ system dysfunction. The purpose of this study is to determine if one year of therapy with iduronate-2-sulfatase enzyme replacement therapy, at a dose of 0.5mg/kg, weekly or every other week, is safe, and results in clinically meaningful improvement in multiple organ function, compared with a placebo group. Upon completion of the study, patients will be eligible to enroll in an open-label maintenance study.

Conditions

• Mucopolysaccharidosis II

Interventions

Type	Name	Description
BIOLOGICAL	Iduronate-2-sulfatase enzyme replacement therapy	Patients will receive weekly infusions of idursulfase at a dose of 0.5 mg/kg.
BIOLOGICAL	iduronate-2-sulfatase enzyme replacement therapy	Patients will receive every other week infusions of idursulfase at a dose of 0.5 mg/kg.
BIOLOGICAL	Placebo	Patients will receive weekly infusions of placebo.

Timeline

• Start date: 2003-09-18
• Primary completion: 2005-03-16
• Completion: 2005-03-16
• First posted: 2003-10-01
• Last updated: 2021-06-10
• Results posted: 2015-05-13

Locations

9 sites across 4 countries: United States, Brazil, Germany, United Kingdom