NCT00741338

Immune Tolerance Study With Aldurazyme® (Laronidase)

A Trial of Antigen-specific Immune Tolerance Induction in Mucopolysaccharidosis I (MPS I) Patients Initiating Enzyme Replacement Therapy With Aldurazyme® (Laronidase)

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 7 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to see if treatment with an antigen-specific immunosuppressive can decrease or stop an antibody response to laronidase (Aldurazyme®) during enzyme replacement therapy with laronidase in severe Mucopolysaccharidosis I (MPS I) participants.

Conditions

• Mucopolysaccharidosis I

Interventions

Type	Name	Description
BIOLOGICAL	Laronidase	0.058 mg/kg - 0.58 mg/kg IV infusion weekly.
DRUG	Cyclosporine A (CsA)	Orally three times daily.
DRUG	Azathioprine (Aza)	Orally either every day for Cohort 1 or every other day for Cohort 2.

Timeline

• Start date: 2008-09-01
• Primary completion: 2012-09-01
• Completion: 2012-09-01
• First posted: 2008-08-26
• Last updated: 2014-07-02
• Results posted: 2014-07-02

Locations

2 sites across 2 countries: Brazil, Russia