Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI
Mucopolysaccharidosis VI
About this trial
This is an interventional treatment trial for Mucopolysaccharidosis VI
Eligibility Criteria
Inclusion Criteria: Patient consent Patient must be five years of age or older Patient must have documented biochemical or genetic proof of MPS VI Patient must walk at least 1 meter, but less than 250 meters, in the first 6 minutes of the baseline 12-minute walk test If female of childbearing potential, patient must have a negative pregnancy test Exclusion Criteria: Patient is under consideration for or has undergone a successful bone marrow transplant (BMT). Pregnant or lactating patient Patient has received an investigational drug within 30 days prior to study enrollment Patient has been previously treated with rhASB Patient has a medical condition, serious intercurrent illness, or other extenuating circumstance that may significantly confound study results or decrease study compliance Patient has a known hypersensitivity to rhASB or to components of the study drug History of cancer (except low grade and fully resolved skin malignancy)
Sites / Locations
- BioMarin Pharmaceutical Inc.