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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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


Sponsor: BioMarin Pharmaceutical

Current Primary Outcome:

  • Change in Height [ Time Frame: 52 weeks ]
  • Change in Weight [ Time Frame: 52 weeks ]
  • Change in Haed Circumference [ Time Frame: 52 weeks ]


Original Primary Outcome: The primary objective of the study is 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

Current Secondary Outcome: Change in Urinary Glycosaminoglycan Levels [ Time Frame: minimum 52 weeks of dosing ]

Change in urinary GAG levels was calculated from baseline to week 52 of treatment.


Original Secondary Outcome:

Information By: BioMarin Pharmaceutical

Dates:
Date Received: March 2, 2006
Date Started: May 2006
Date Completion:
Last Updated: July 19, 2011
Last Verified: July 2011