Clinical Trial: Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title:
Brief Summary: 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.
Detailed Summary:
Sponsor: BioMarin Pharmaceutical
Current Primary Outcome: Change From Baseline in 12-minute Walk Test at 24 Weeks [ Time Frame: baseline and 24 weeks ]
Original Primary Outcome: Lysosomal Storage Disease
Current Secondary Outcome:
- 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 ]
Original Secondary Outcome:
Information By: BioMarin Pharmaceutical
Dates:
Date Received: August 20, 2003
Date Started: September 2003
Date Completion: March 2004
Last Updated: December 18, 2009
Last Verified: December 2009
