A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
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|ClinicalTrials.gov Identifier: NCT01275066|
Recruitment Status : Completed
First Posted : January 12, 2011
Results First Posted : April 15, 2014
Last Update Posted : July 7, 2014
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.
|Condition or disease||Intervention/treatment||Phase|
|MPS IV A||Drug: BMN 110 Weekly Drug: Placebo Drug: BMN 110 Every Other Week||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||177 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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)|
|Study Start Date :||February 2011|
|Actual Primary Completion Date :||August 2012|
|Actual Study Completion Date :||August 2012|
|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.
- 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 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01275066
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|Study Director:||Debra Lounsbury||BioMarin Pharmaceutical|