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Open-Label Study of Efficacy and Safety 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.
 
ClinicalTrials.gov Identifier: NCT00048711
Recruitment Status : Completed
First Posted : November 8, 2002
Last Update Posted : August 25, 2009
Sponsor:
Information provided by:
BioMarin Pharmaceutical

Brief Summary:
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 or disease Intervention/treatment Phase
Mucopolysaccharidosis VI Drug: N-acetylgalactosamine 4-sulfatase Phase 2

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Study Type : Interventional  (Clinical Trial)
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (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
Study Start Date : March 2002
Actual Study Completion Date : June 2006





Primary Outcome Measures :
  1. 12-minute walk test [ Time Frame: weeks 6, 12, 24, 48, 96 and 144 ]
  2. urinary glycoaminoglycan(uGAG)levels [ Time Frame: weeks 1, 4, 6, 8, 12 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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)

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 ClinicalTrials.gov identifier (NCT number): NCT00048711


Locations
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United States, California
BioMarin Pharmaceutical Inc.
Novato, California, United States, 94949
Sponsors and Collaborators
BioMarin Pharmaceutical
Investigators
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Study Director: Stuart J Sweidler, MD, Ph.D. BioMarin Pharmaceutical
Additional Information:
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ClinicalTrials.gov Identifier: NCT00048711    
Other Study ID Numbers: ASB-01-04
First Posted: November 8, 2002    Key Record Dates
Last Update Posted: August 25, 2009
Last Verified: August 2009
Additional relevant MeSH terms:
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Mucopolysaccharidoses
Mucopolysaccharidosis VI
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases