NCT00299000

A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI

A Phase 4 Multi-center, Multi-national, Open-label, Randomized, Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With Maroteaux-Lamy Syndrome (MPS VI)

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 4 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 1 Year
• Healthy volunteers: Not accepted

Summary

The purpose of the study is to evaluate the safety and efficacy of two dose levels of Naglazyme in infants under the age of one year who have MPS VI by monitoring physical appearance, x-ray of the skeletal system and growth.

Detailed description

The primary objective of the study was to evaluate the efficacy of two dose levels of Naglazyme in preventing the progression of skeletal dysplasia in infants under the age of one year who have MPS VI by monitoring physical appearance, x-ray of the skeletal system and growth. The secondary objective of the study was to evaluate the efficacy of the two dose levels of Naglazyme in preventing several measures of disease progression in infants under the age of one year who have MPS VI by monitoring urinary GAGs, gross and fine motor function, cardiac function, vision, hearing, and use of health resources. The safety objective of the study was to evaluate the safety of two dose levels of Naglazyme in infants under the age of one year who have MPS VI.

Conditions

• Mucopolysaccharidosis VI
• Maroteaux-Lamy Syndrome

Interventions

Type	Name	Description
DRUG	Naglazyme	Weekly infusion for minimum of 52 weeks. Naglazyme is diluted in sterile 0.9% sodium chloride solution

Timeline

• Start date: 2006-05-01
• Primary completion: 2009-04-01
• Completion: 2009-04-01
• First posted: 2006-03-06
• Last updated: 2011-07-22
• Results posted: 2010-10-15

Locations

4 sites across 3 countries: United States, France, Portugal