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

This study has been completed.
Information provided by:
BioMarin Pharmaceutical Identifier:
First received: November 4, 2002
Last updated: November 2, 2006
Last verified: November 2006

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 Intervention Phase
Mucopolysaccharidosis VI
Drug: N-acetylgalactosamine 4-sulfatase
Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double-Blind
Primary Purpose: Treatment
Official Title: Double-Blind,2 Dose Group Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI

Resource links provided by NLM:

Further study details as provided by BioMarin Pharmaceutical:

Primary Outcome Measures:
  • Lysosomal storage disease

Study Start Date: September 2000
Estimated Study Completion Date: November 2005

Genders Eligible for Study:   Both
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)
  Contacts and Locations
Please refer to this study by its identifier: NCT00048620

United States, California
BioMarin Pharmaceutical Inc.
Novato, California, United States, 94949
Sponsors and Collaborators
BioMarin Pharmaceutical
Study Director: Stuart J Swiedler, MD, Ph.D. BioMarin Pharmaceutical
  More Information

Additional Information:
No publications provided Identifier: NCT00048620     History of Changes
Other Study ID Numbers: ASB-00-01
Study First Received: November 4, 2002
Last Updated: November 2, 2006
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Mucopolysaccharidosis VI
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Connective Tissue Diseases
Metabolic Diseases processed this record on April 16, 2014