Clinical Trial: Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI

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)

Detailed Summary:
Sponsor: BioMarin Pharmaceutical

Current Primary Outcome:

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


Original Primary Outcome:

Current Secondary Outcome:

Original Secondary Outcome:

Information By: BioMarin Pharmaceutical

Dates:
Date Received: November 6, 2002
Date Started: March 2002
Date Completion:
Last Updated: August 24, 2009
Last Verified: August 2009