aDesign of pivotal trial, and pivotal extension trial.1,2
The pivotal trial was a randomised (1:1:1), double-blind, placebo-controlled, 53-week phase II/III trial. The study population included treatment-naïve male MPS II patients, aged 5–31 years (N=96).
Three patient groups were compared in this study:
ELAPRASE IV 0.5 mg/kg weekly (n=32)
ELAPRASE IV 0.5 mg/kg every other week (n=32)
The primary endpoint was a two-component composite score based on the sum of the ranks of the change from baseline to week 53 in 6MWT as a measure of endurance and %FVC as a measure of pulmonary function. Secondary endpoints included: 6MWT distance, %FVC, absolute FVC, liver and spleen volumes, uGAG excretion, and passive JROM. Safety assessments included: physical exam, serum chemistry, complete blood count, urinalysis, measurement of vital signs, height and weight, and ECG, AEs were monitored and recorded throughout the study
The pivotal extension trial was an open-label, 24-month extension trial for patients, aged 5–31 years, who completed the 53-week Phase II/III pivotal trial (N=94). The primary outcome measures were safety, 6MWT distance, and FVC. The secondary outcome measures included liver, and spleen volume, uGAG excretion, cardiac mass, JROM, linear growth velocity, and functional status. Safety assessments included: clinical assessment of treatment safety at each study visit. AEs were monitored and recorded throughout the study. Presence of anti-idursulfase antibodies was assayed.