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.
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
PREVENTION
Masking
NONE
Enrollment
4
Weekly infusion for minimum of 52 weeks. Naglazyme is diluted in sterile 0.9% sodium chloride solution
Children's Hospital Los Angeles
Los Angeles, California, United States
Children's Hospital Oakland
Oakland, California, United States
Hospital Femme Mere Enfant Centre
Lyon, France
Hospital PediAtrico de Coimbra
Coimbra, Portugal
Change in Height
Time frame: 52 weeks
Change in Weight
Time frame: 52 weeks
Change in Haed Circumference
Time frame: 52 weeks
Change in Urinary Glycosaminoglycan Levels
Change in urinary GAG levels was calculated from baseline to week 52 of treatment.
Time frame: minimum 52 weeks of dosing
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