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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
39
BioMarin Pharmaceutical Inc.
Novato, California, United States
Change From Baseline in 12-minute Walk Test at 24 Weeks
Number of meters walked in 12 minutes at week 24 minus number of meters walked in 12 minutes at baseline
Time frame: baseline and 24 weeks
Change From Baseline in 3-minute Stair Climb at 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
Time frame: baseline and 24 weeks
Change From Baseline in Urinary GAG (uGAG) at 24 Weeks
Glycosaminoglycan (GAG) level measured in urine
Time frame: baseline and 24 weeks
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