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Novato, California 94949


The purpose of the study is to evaluate the safety, efficacy and pharmacokinetics of two dose levels of weekly intravenous infusions of recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) for a minimum of 24 weeks in patients diagnosed with MPS VI.


Inclusion Criteria: - Patient consent - Patient must be five years of age or older - Patient must have documented diagnosis of MPS VI, confirmed at screening by measurable clinical signs and symptoms of MPS VI - Leukocyte ASB enzyme activity level less than 20% of the normal range - Clinical evidence of significant MPS VI disease that provides adequate opportunity to achieve quantitative, short-term therapeutic benefit in three or more of the following parameters: endurance (as measured by a six-minute walk test), forced vital capacity (as measured by spirometry), joint range of motion, urinary glycosaminoglycans, and hepatomegaly. - Ability to perform all protocol tests - Ability to stand independently for six minutes - Sexually active subjects must agree to use an adequate form of contraception Exclusion Criteria: - History of bone marrow transplantation - Pregnant or lactating patient - Use of an investigational drug or device within 30 days prior to study participation. - A medical condition, serious intercurrent illness, or other extenuating circumstances that may significantly decrease study compliance including prescribed follow-up - 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 Swiedler, 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: ClinicalTrials.gov

Date Processed: October 09, 2019

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