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Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00048620
Recruitment Status : Completed
First Posted : November 6, 2002
Last Update Posted : November 3, 2006
Information provided by:
BioMarin Pharmaceutical

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Mucopolysaccharidosis VI Drug: N-acetylgalactosamine 4-sulfatase Phase 1

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Study Type : Interventional  (Clinical Trial)
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double
Primary Purpose: Treatment
Official Title: Double-Blind,2 Dose Group Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI
Study Start Date : September 2000
Study Completion Date : November 2005

Primary Outcome Measures :
  1. Lysosomal storage disease

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00048620

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United States, California
BioMarin Pharmaceutical Inc.
Novato, California, United States, 94949
Sponsors and Collaborators
BioMarin Pharmaceutical
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Study Director: Stuart J Swiedler, MD, Ph.D. BioMarin Pharmaceutical
Additional Information:
Layout table for additonal information Identifier: NCT00048620    
Other Study ID Numbers: ASB-00-01
First Posted: November 6, 2002    Key Record Dates
Last Update Posted: November 3, 2006
Last Verified: November 2006
Additional relevant MeSH terms:
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Mucopolysaccharidosis VI
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Connective Tissue Diseases
Metabolic Diseases