NCT00146757

A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old

A Phase II Open-Label Clinical Trial of Recombinant Human Alpha-L-iduronidase (Aldurazyme®) to Evaluate the Safety and Pharmacokinetics in Mucopolysaccharidosis I (MPS I) Patients Less Than 5 Years Old

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

The main objectives of this study are to evaluate the safety and pharmacokinetics (PK) of enzyme replacement therapy with recombinant human alpha-L-iduronidase \[Aldurazyme® (laronidase)\] in mucopolysaccharidosis I (MPS I) patients less than 5 years old. Efficacy measurements will also be evaluated in this study.

Conditions

• Mucopolysaccharidosis I
• Hurler Syndrome
• Hurler-Scheie Syndrome
• Scheie Syndrome

Interventions

Type	Name	Description
BIOLOGICAL	Aldurazyme (Recombinant Human Alpha-L-Iduronidase)	100 U/kg every week
BIOLOGICAL	Aldurazyme (Recombinant Human Alpha-L-Iduronidase)	200 U/kg every week (Week 26 onwards)

Timeline

• Start date: 2002-10-01
• Primary completion: 2005-05-01
• Completion: 2005-05-01
• First posted: 2005-09-07
• Last updated: 2015-04-03
• Results posted: 2009-06-16

Locations

4 sites across 4 countries: France, Germany, Netherlands, United Kingdom