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 MOR-006 CPET Limited ambulation Grip/ Pinch
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 16
Healthy volunteers: no

Brief summary:
The primary objective of this study is to evaluate the effect of 2.0 mg/kg/week BMN 110 in a patient population that has limited ambulation, in a period of up to 144 weeks.

Detailed description:
Effect is defined by the following key domains:

* Upper extremity function and dexterity
* Mobility

Arms:
arm 1: Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion for up to 144 weeks.

Interventions: intervention 1: BMN 110
Intervention descriptions: intervention 1: Drug will be delivered through a 4 hour (approximate) IV infusion at a dosage amount of 2.0 mg/kg/week for up to 144 weeks of treatment.

Protocol document: