A Double-Blind Study to Evaluate the Efficacy and Safety of 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.
|MPS IV A||Drug: BMN 110 Weekly Drug: Placebo Drug: BMN 110 Every Other Week||Phase 3|
|Study Design:||Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|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)|
- 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 ]
|Study Start Date:||February 2011|
|Study Completion Date:||August 2012|
|Primary Completion Date:||August 2012 (Final data collection date for primary outcome measure)|
|Placebo Comparator: Placebo||
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.
|Experimental: BMN 110 Weekly||
Drug: BMN 110 Weekly
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.
|Experimental: BMN 110 Every Other Week||
Drug: BMN 110 Every Other 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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01275066
Show 28 Study Locations
|Study Director:||Debra Lounsbury||BioMarin Pharmaceutical|