Clinical Trial: A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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 Muco

Brief 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.


Detailed Summary:
Sponsor: BioMarin Pharmaceutical

Current Primary Outcome: Change From Baseline in Endurance as Measured by the 6-minute Walk Test [ Time Frame: Baseline to Week 24 ]

Original Primary Outcome: Change From Baseline in Endurance as Measured by the 6-minute Walk Test [ Time Frame: Screening and every 12 weeks ]

Current Secondary Outcome:

  • 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 ]


Original Secondary Outcome:

  • Change From Baseline in Endurance as Measured by the 3-minute Stair Climb Test [ Time Frame: Screening and every 12 weeks ]
  • Change from baseline in urine keratan sulfate (KS) levels (normalized to creatinine) [ Time Frame: Baseline, week 2, 4, 8, 12, 16, 20, and 24 ]


Information By: BioMarin Pharmaceutical

Dates:
Date Received: January 10, 2011
Date Started: February 2011
Date Completion:
Last Updated: June 6, 2014
Last Verified: June 2014