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 at the time of administration of the first dose of study drug, diagnosed with MPS IVA (Morquio A Syndrome) for up to 208 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 208 weeks/ETV ] [ 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, 52, 78, 104, 130, 156, 182, 208/ETV ] [ Designated as safety issue: No ]
- Change in patient growth over time [ Time Frame: Baseline and Weeks: 13, 26, 39, 52,78, 104, 130, 156, 182, 208/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:||October 2011|
|Estimated Study Completion Date:||June 2015|
|Estimated Primary Completion Date:||June 2015 (Final data collection date for primary outcome measure)|
|Experimental: BMN 110 Weekly||
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 208 weeks.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01515956
|United States, California|
|Oakland, California, United States|
|United States, New York|
|Manhasset, New York, United States|
|Central Manchester, United Kingdom|
|Study Director:||Adam Shaywitz, MD, PhD||BioMarin Pharmaceutical|