Conditions: Mucopolysaccharidosis IVA Morquio A Syndrome MPS IVA
Keywords: Mucopolysaccharidosis IVA type A MPS IVA Type A Mucopolysaccharidosis IVA MPS IVA Morquio A Syndrome Lysosomal Storage Disorder LSD N-acetylgalactosamine-6-sulfatase N-acetylgalactosamine-6-sulfate sulfatase galactose-6-sulfatase GALNS enzyme replacement therapy ERT
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 15
Healthy volunteers: no

Brief summary:
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.

Detailed description:

Arms:
arm 1: None

Interventions: intervention 1: BMN 110
Intervention descriptions: intervention 1: 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.

Protocol document: