Conditions: MPS IV A Mucopolysaccharidosis IVA Morquio A Syndrome
Keywords: Mucopolysaccharidosis IV type A MPS IV 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: 20
Healthy volunteers: no

Brief summary:
This multicenter, open-label extension study is designed to assess long-term efficacy and safety of 2.0 milligrams (mg)/kilogram(kg)/week of BMN 110 in patients diagnosed with Mucopolysaccharidosis IVA (MPS IVA). Patients with MPS IVA, who enrolled in a prior BioMarin sponsored clinical study of BMN 110 (NCT00884949; Study Identification Number MOR-002), were eligible to enroll in this study (except patients who enrolled in NCT01275066; Study Identification Number MOR-004).

Detailed description:

Arms:
arm 1: None

Interventions: intervention 1: BMN 110
Intervention descriptions: intervention 1: Patients will receive an intravenous infusion of BMN110 at 2.0mg/kg/week, over a period of approximately 4 hours per infusion, for up to 240 weeks.

Protocol document: