MPS IV A Clinical Trial
Official title:
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/Week and 2.0 mg/kg/Every Other Week BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
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.
n/a
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
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Terminated |
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Phase 1/Phase 2 | |
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