NCT00912925

Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I

A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Multinational, Clinical Study of Recombinant Human Alpha L-Iduronidase In Patients With Mucopolysaccharidosis I

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 45 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

This study is being conducted to demonstrate the safety and clinical efficacy of Aldurazyme treatment in MPS I patients

Conditions

• Mucopolysaccharidosis I
• Hurlers Syndrome
• Hurler-Scheie Syndrome

Interventions

Type	Name	Description
BIOLOGICAL	rhIDU (recombinant human-Alpha-L-Iduronidase)	Patients in the active treatment group received Aldurazyme intravenously at a dose of 100 units/kg (approximately 0.58mg/kg) administered intravenously over approximately 4 hours once weekly for 26 weeks.
BIOLOGICAL	Placebo	Patients in the Placebo-control group were administered a solution of 100mM sodium phosphate , 150mM sodium chloride, and 0.001% polysorbate-80, adjusted to a pH of 5.8 administered intravenously over a time period of approximately 4 hours once weekly for 26 weeks.

Timeline

• Start date: 2000-12-01
• Primary completion: 2001-09-01
• Completion: 2001-09-01
• First posted: 2009-06-03
• Last updated: 2015-04-07
• Results posted: 2009-08-19

Locations

4 sites across 3 countries: United States, Canada, Germany