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Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI

This study has been completed.
Information provided by:
BioMarin Pharmaceutical Identifier:
First received: August 20, 2003
Last updated: December 18, 2009
Last verified: December 2009
The purpose of the study is to evaluate the ability of rhASB versus placebo to enhance endurance in patients with Mucopolysaccharidosis VI (MPS VI), as evidenced by an increase in the number of meters walked in the 12 minute walk test at Week 24 compared with baseline.

Condition Intervention Phase
Mucopolysaccharidosis VI Drug: Placebo Drug: N-acetylgalactosamine 4-sulfatase Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double
Primary Purpose: Treatment

Resource links provided by NLM:

Further study details as provided by BioMarin Pharmaceutical:

Primary Outcome Measures:
  • Change From Baseline in 12-minute Walk Test at 24 Weeks [ Time Frame: baseline and 24 weeks ]

Secondary Outcome Measures:
  • Change From Baseline in 3-minute Stair Climb at 24 Weeks [ Time Frame: baseline and 24 weeks ]
  • Change From Baseline in Urinary GAG (uGAG) at 24 Weeks [ Time Frame: baseline and 24 weeks ]

Enrollment: 39
Study Start Date: September 2003
Estimated Study Completion Date: March 2004
Arms Assigned Interventions
Placebo Comparator: Placebo Drug: Placebo
Active Comparator: rhASB Drug: N-acetylgalactosamine 4-sulfatase
Other Name: rhASB


Ages Eligible for Study:   7 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria

  • Patient consent
  • Patient must be seven years of age or older
  • Patient must have documented biochemical or genetic proof of MPS VI
  • The patient must be able to walk independently at least 5 meters and no more than 270 meters in the first 6 minutes, or no more then 400 meters total in 12 minutes, in the screening 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)
  • Patient refuses or is unable to complete all screening evaluations
  • 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
  • The patient has clinically significant spinal cord compression
  • The patient has known hypersensitivity to rhASB or to components of the active or placebo test solutions
  Contacts and Locations
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Please refer to this study by its identifier: NCT00067470

United States, California
BioMarin Pharmaceutical Inc.
Novato, California, United States, 94949
Sponsors and Collaborators
BioMarin Pharmaceutical
  More Information

Additional Information: Identifier: NCT00067470     History of Changes
Other Study ID Numbers: ASB-03-05
Study First Received: August 20, 2003
Results First Received: February 4, 2009
Last Updated: December 18, 2009

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 September 20, 2017