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).
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
20
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.
Unnamed facility
Birmingham, United Kingdom
Unnamed facility
London, United Kingdom
Unnamed facility
Manchester, United Kingdom
Subject Incidence of Treatment Emergent AEs
The primary objective of the study was to evaluate the safety of weekly infusions of BMN 110 administered in escalating doses to subjects with MPS IVA. The safety variable incidence of TEAE is summarized.
Time frame: Entire Study, through week 84
Change From Baseline in 6MWT
Change from baseline in meters in 6-minute Walk Test. As a measure of endurance, a 6-minute walk test (6MWT) was performed according to the American Thoracic Society Guidelines. Patients were instructed to walk as far as possible in 6 minutes.
Time frame: Baseline to Weeks 12, 24, 36, 48, 72
Change From Baseline in 3MSCT
Change from baseline in the 3-minute Stair Climb Test. Patients walked up stairs that have a railing, which could be used for support, for 3 minutes, with the number of stairs climbed recorded. The test result was the number of steps climbed per minute.
Time frame: Baseline to Weeks 12, 24, 36, 48, 72
Percent Change From Baseline in uKS
Percent Change from baseline in Normalized Urine KS. The percent change was calculated (Week X value - baseline value)/baseline value \*100%
Time frame: Baseline to Weeks 12, 24, 36, 72
Percent Change From Baseline in MVV
Percent Change from baseline in Maximum Voluntary Ventilation.
Time frame: Baseline to Weeks 12, 24, 36, 72
Percent Change From Baseline in FVC
Percent Change from baseline in Forced Vital Capacity.
Time frame: Baseline to Weeks 12, 24, 36, 72
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