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Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase (rhASB) in Patients With MPS VI
This study has been completed.
Sponsored by: BioMarin Pharmaceutical
Information provided by: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT00067470
  Purpose

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: N-acetylgalactosamine 4-sulfatase
Phase III

U.S. FDA Resources
Study Type: Interventional
Study Design: Treatment, Randomized, Double-Blind, Placebo Control, Parallel Assignment, Safety/Efficacy Study

Further study details as provided by BioMarin Pharmaceutical:

Primary Outcome Measures:
  • Lysosomal Storage Disease

Study Start Date: September 2003
Estimated Study Completion Date: March 2004
  Eligibility

Ages Eligible for Study:   7 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
Please refer to this study by its ClinicalTrials.gov identifier: NCT00067470

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

Related Info  This link exits the ClinicalTrials.gov site

Study ID Numbers: ASB-03-05
Study First Received: August 20, 2003
Last Updated: June 23, 2005
ClinicalTrials.gov Identifier: NCT00067470  
Health Authority: United States: Food and Drug Administration

Study placed in the following topic categories:
Metabolism, Inborn Errors
Mucopolysaccharidoses
Metabolic Diseases
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Connective Tissue Diseases
Mucopolysaccharidosis
Metabolic disorder
Mucopolysaccharidosis VI

Additional relevant MeSH terms:
Mucinoses
Carbohydrate Metabolism, Inborn Errors

ClinicalTrials.gov processed this record on January 14, 2009