Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
This trial has been completed.
|Conditions||mucopolysaccharidosis iva, morquio a syndrome, mps iva|
|Start date||October 2011|
|End date||February 2016|
|Trial size||15 participants|
|Trial identifier||NCT01515956, MOR-007|
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.
|United States||No locations recruiting|
|Other countries||No locations recruiting|
|Oakland, CA||not available||completed|
|Manhasset, NY||not available||completed|
|Monza, Italy||not available||completed|
|Taipei, Taiwan||not available||completed|
|Central Manchester, United Kingdom||not available||completed|
|Endpoint classification||safety/efficacy study|
|Intervention model||single group assignment|
Descriptive summary of clinical safety assessments
time frame: Up to 208 weeks/ETV
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
Change in patient growth over time
time frame: Baseline and Weeks: 13, 26, 39, 52,78, 104, 130, 156, 182, 208/ETV
Male or female participants up to 5 years old.
Inclusion Criteria: - Less than 5 years of age at the time of the first study drug infusion - Documented clinical diagnosis of MPS IVA based on clinical signs and symptoms of MPS IVA and documented reduced fibroblast or leukocyte GALNS enzyme activity or genetic testing confirming diagnosis of MPS IVA - Written informed consent provided by parent or legally authorized representative after the nature of the study has been explained and prior to any research-related procedures. Exclusion Criteria: - Previous hematopoietic stem cell transplant (HSCT). - Previous treatment with BMN 110. - Known hypersensitivity to any of the components of BMN 110. - Major surgery within 3 months prior to stuy entry or planned major surgery during the 52-week treatment period. - Use of any investigational product or investigational medical device within 30 days prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments. - Concurrent disease or condition, including but not limited to symptomatic cervical spine instability, clinically significant spinal cord compression, or severe cardiac disease that would interfere with study participation or safety as determined by the Investigator. - Any condition that, in the view of the Investigator, places the patient at high risk of poor treatment compliance or of not completing the study.
|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)|
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