A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Summary
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.
Timeline
- Start
- 2011-02
- Primary completion
- 2012-08
- Completion
- 2012-08
Outcome
Mixed primary results
registry analysis (superiority or other test); Placebo vs BMN110 2.0 mg/kg/Week; p = 0.0174; Mean Difference (Final Values) 22.5; ANCOVA NCT01275066 ↗
registry MEAN BMN110 2.0 mg/kg/Week: Change from Baseline to Week 24: 36.0 (Standard Deviation 58.11) meters NCT01275066 ↗
registry analysis (superiority or other test); Placebo vs BMN110 2.0 mg/kg/Qow; p = 0.9542; Mean Difference (Final Values) 0.5; ANCOVA NCT01275066 ↗
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | elosulfase alfa | Protein / enzyme biologic | 2 mg/kg | Intravenous |