Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
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|ClinicalTrials.gov Identifier: NCT01515956|
Recruitment Status : Completed
First Posted : January 24, 2012
Results First Posted : July 6, 2017
Last Update Posted : August 10, 2017
|Condition or disease||Intervention/treatment||Phase|
|Mucopolysaccharidosis IVA Morquio A Syndrome MPS IVA||Drug: BMN 110||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||15 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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)|
|Study Start Date :||October 2011|
|Actual Primary Completion Date :||February 2016|
|Actual Study Completion Date :||February 2016|
|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.
- 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 [ Time Frame: 52 weeks ]Number of Participants Experiencing Adverse Events
- Percent Change From Baseline to Week 52 in Urinary Keratan Sulfate Measures [ Time Frame: Baseline to Week 52 ]Percent Change from Baseline to Week 52 for Urinary Keratan Sulfate measures.
- Change From Baseline in Normalized Growth Rate Z-Scores [ Time Frame: Baseline to Week 52 ]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).
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01515956
|United States, California|
|Oakland, California, United States|
|United States, New York|
|Manhasset, New York, United States|
|Central Manchester, United Kingdom|
|Study Director:||Debra Lounsbury||BioMarin Pharmaceutical|