This Phase 3 study will evaluate the efficacy and safety of 2.0 mg/kg/week BMN 110 and 2.0 mg/kg/every other week BMN 110 in patients with mucopolysaccharidosis IVA (Morquio A Syndrome). There is currently no standard accepted treatment for MPS IVA other than supportive care. Enzyme replacement therapy (ERT) may be a potential new treatment option for MPS IVA patients. BMN 110 is administered to MPS IVA patients by IV infusion, allowing cellular uptake by the mannose-6-phosphate receptor and transportation to the lysosomes. This enzyme uptake into the lysosomes is hypothesized to promote increased catabolism of keratan sulfate (KS) in tissue macrophages, hyaline cartilage, other connective tissues, and heart valve, and reduce the progressive accumulation of KS which is responsible for the clinical manifestations of the disorders.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
177
BMN 110 Weekly: Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours once a week.
Intravenous infusion of placebo solution at a volume equivalent to that needed for 2.0 mg/kg dose of BMN 110 administered over a period of approximately 4 hours once a week.
BMN 110 Every Other Week: Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours every other week and infusions of placebo on alternating weeks.
Unnamed facility
Oakland, California, United States
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Wilmington, Delaware, United States
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Washington D.C., District of Columbia, United States
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Chicago, Illinois, United States
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New York, New York, United States
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Seattle, Washington, United States
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Córdoba, Argentina
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Campina Grande, Brazil
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Porto Alegre, Brazil
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Montreal, Canada
...and 18 more locations
Change From Baseline in Endurance as Measured by the 6-minute Walk Test
Time frame: Baseline to Week 24
Change From Baseline in Endurance as Measured by the 3-minute Stair Climb Test
Time frame: Baseline to Week 24
Percent Change From Baseline in Urine Keratan Sulfate Normalized for Urine Creatinine
Time frame: Baseline to Week 24
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