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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
15
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.
Unnamed facility
Oakland, California, United States
Unnamed facility
Manhasset, New York, United States
Unnamed facility
Monza, Italy
Unnamed facility
Taipei, Taiwan
Unnamed facility
Central Manchester, United Kingdom
To Evaluate Safety and Tolerability of Infusions of BMN 110 at a Dose of 2.0 mg/kg/Week Over a 52-week Period in MPS IVA Subjects Less Than 5 Years of Age at Time of First Study Drug Infusion
Number of Participants Experiencing Adverse Events
Time frame: 52 weeks
Percent Change From Baseline to Week 52 in Urinary Keratan Sulfate Measures
Percent Change from Baseline to Week 52 for Urinary Keratan Sulfate measures.
Time frame: Baseline to Week 52
Change From Baseline in Normalized Growth Rate Z-Scores
Changes in growth over time will be assessed using anthropometric measurements and radiographs of lower extremities. Z-scores are the normalized scores derived from the reference population mean and standard deviation (A positive change from baseline indicates that the population has moved closer to the reference population and represents a positive outcome).
Time frame: Baseline to Week 52
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.