Mucopolysaccharidosis VI Clinical Trial
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 (MPS VI)
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.
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Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Prevention
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