Novato, California 94949

  • Mucopolysaccharidosis VI


The purpose of the study is evaluate the efficacy, safety, and pharmacokinetics of weekly intravenous infusions of 1 mg/kg recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) in patients diagnosed with Mucopolysaccharidosis VI (MPS VI)


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)



Primary Contact:

Study Director
Stuart J Sweidler, MD, Ph.D.
BioMarin Pharmaceutical

Backup Contact:


Location Contact:

Novato, California 94949
United States

There is no listed contact information for this specific location.

Site Status: N/A

Data Source:

Date Processed: March 26, 2020

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