A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI
Mucopolysaccharidosis VI, Maroteaux-Lamy Syndrome
About this trial
This is an interventional prevention trial for Mucopolysaccharidosis VI
Eligibility Criteria
Inclusion Criteria: Signed informed consent by a parent or legal guardian Parent or legal guardian willing and able to comply with all study procedures Equal to or greater than 36 weeks estimated gestational age by physical exam at birth Has a diagnosis of MPS VI based on a documented prenatal diagnosis or fibroblast or leukocyte N-acetylgalactosamine 4-sulfatase (ASB) enzyme activity level of less than 10% of the lower limit of the normal range of the measuring laboratory Is less than one year of age Has no evidence of skeletal dysplasia based on physical exam Exclusion Criteria: Parent of legal guardian perceived to be unreliable or unavailable for study participation Use of any investigational drug within 30 days prior to screening, or requirement for any investigational agent prior to completion of all scheduled study assessments Has concurrent disease or condition that would interfere with study participation or safety (i.e., has previously undergone hematopoietic stem cell transplantation such as bone marrow or cord blood transplantation, or major organ transplantation) Any condition that, in the view of the principle investigator, renders the subject at high risk from treatment compliance and/or completing the study Has known hypersensitivity to Naglazyme Has previously received Naglazyme
Sites / Locations
- Children's Hospital Los Angeles
- Children's Hospital Oakland
- Hospital Femme Mere Enfant Centre
- Hospital PediAtrico de Coimbra
Arms of the Study
Arm 1
Arm 2
Other
Other
Naglazyme, 1.0 mg/kg
Naglazyme, 2.0 mg/kg
Dose comparison
Dose Comparison