Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI
Phase 3
Completed
- Conditions
- Mucopolysaccharidosis VI
- Interventions
- Drug: N-acetylgalactosamine 4-sulfataseDrug: Placebo
- Registration Number
- NCT00067470
- Lead Sponsor
- BioMarin Pharmaceutical
- 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 Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 39
Inclusion Criteria
Not provided
Exclusion Criteria
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description rhASB N-acetylgalactosamine 4-sulfatase - Placebo Placebo -
- Primary Outcome Measures
Name Time Method Change From Baseline in 12-minute Walk Test at 24 Weeks baseline and 24 weeks Number of meters walked in 12 minutes at week 24 minus number of meters walked in 12 minutes at baseline
- Secondary Outcome Measures
Name Time Method Change From Baseline in 3-minute Stair Climb at 24 Weeks baseline and 24 weeks Number of stairs climbed per minute in 3 minutes at 24 weeks minus number of stairs climbed per minute in 3 minutes at baseline
Change From Baseline in Urinary GAG (uGAG) at 24 Weeks baseline and 24 weeks Glycosaminoglycan (GAG) level measured in urine
Trial Locations
- Locations (1)
BioMarin Pharmaceutical Inc.
🇺🇸Novato, California, United States