Open-Label Study of Efficacy and Safety 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 6, 2002
Last updated: August 24, 2009
Last verified: August 2009

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)

Condition Intervention Phase
Mucopolysaccharidosis VI
Drug: N-acetylgalactosamine 4-sulfatase
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Open-Label Study of Efficacy and Safety 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:
  • 12-minute walk test [ Time Frame: weeks 6, 12, 24, 48, 96 and 144 ] [ Designated as safety issue: No ]
  • urinary glycoaminoglycan(uGAG)levels [ Time Frame: weeks 1, 4, 6, 8, 12 ] [ Designated as safety issue: No ]

Study Start Date: March 2002
Study Completion Date: June 2006

Ages Eligible for Study:   5 Years and older
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 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)
  Contacts and Locations
Please refer to this study by its identifier: NCT00048711

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

Additional Information:
No publications provided Identifier: NCT00048711     History of Changes
Other Study ID Numbers: ASB-01-04
Study First Received: November 6, 2002
Last Updated: August 24, 2009
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 20, 2014