Conditions: MPS IV A
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: NON_RANDOMIZED | Intervention model: SINGLE_GROUP
Enrollment: 20
Healthy volunteers: no

Brief summary:
This multicenter, open-label study is designed to assess safety, dose-response using pharmacokinetic (PK) and pharmacodynamic (PD) measures, and clinical efficacy of BMN 110 in subjects between 5 and 18 years of age, diagnosed with Mucopolysaccharidosis IVA (MPS IVA).

Detailed description:

Arms:
arm 1: Within-patient Dose-Escalation

Interventions: intervention 1: BMN 110
Intervention descriptions: intervention 1: Subjects will receive a weekly 4- to 5-hour intravenous infusion of BMN 110 in 3 consecutive 12-week dosing intervals, using the following regimen:

* Weeks 1-12: 0.1 mg/kg/week
* Weeks 13-24: 1.0 mg/kg/week
* Weeks 25-36: 2.0 mg/kg/week

Subjects who complete the 36-week Dose-Escalation Period will have the option to continue drug treatment for an additional 36 to 48 weeks. Subjects continuing on treatment after the Dose-Escalation period will receive weekly 4- to 5-hour intravenous infusions of BMN 110 at a dose of 1.0 mg/kg/week.

Protocol document: