Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
This open-label Phase 2 study will evaluate the safety and efficacy of weekly 2.0 mg/kg/wk infusions of BMN 110 in pediatric patients, less than 5 years of age, diagnosed with MPS IVA (Morquio A Syndrome) for 52 weeks.
Morquio A Syndrome
Drug: BMN 110
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase 2, Open-label, Multinational Clinical Study to Evaluate the Safety and Efficacy of BMN 110 in Pediatric Patients Less Than 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)|
- Descriptive summary of clinical safety assessments [ Time Frame: Up to 52 weeks ] [ Designated as safety issue: Yes ]
Safety will be determined by the following factors:
Number and severity of adverse events in participants. Clinically significant changes in any of the following assessments from Baseline: Vital Signs, echocardiogram, ECG, immunogenicity tests, physical and neurological examinations, standard clinical laboratory tests, concomitant medications, and cervical spine radiography.
- Change in Urinary Keratan Sulfate measures over time [ Time Frame: Baseline, and weeks: 2, 4, 8, 13, 26, 39, and 52/ETV ] [ Designated as safety issue: No ]To Evaluate the ability of 2.0 mg/kg/week BMN 110 to reduce the urinary KS levels in MPS IVA patients less than 5 years.
- Change in patient growth over time [ Time Frame: Baseline and Weeks: 13, 26, 39, and 52/ETV ] [ Designated as safety issue: No ]Changes in growth over time will be assessed using anthropometric measurements and radiographs of lower extremities.
|Study Start Date:||November 2011|
|Estimated Study Completion Date:||January 2014|
|Estimated Primary Completion Date:||August 2013 (Final data collection date for primary outcome measure)|
Experimental: BMN 110 Weekly
All enrolled patients will receive IV infusions of study drug at a dose of 2.0 mg/kg/wk administered over a period of approximately 4 hours every week for up to 52 weeks.
Drug: BMN 110
Patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg/wk over a period of approximately 4 hours every week for up to 52 weeks.
|United States, California|
|Oakland, California, United States|
|United States, New York|
|Manhasset, New York, United States|
|Central Manchester, United Kingdom|
|Study Director:||Terence Eagleton, MB BS, BSc.||BioMarin Pharmaceutical|