Primary endpoints | |
Safety of idursulfase • Occurrence of infusion-related reactions and other adverse events (including serious adverse events) • Immunogenicity, as determined by time to first positive antibody response (antibody level and isotype), antibody titre, isotype, and neutralizing antibodies Effectiveness of idursulfase • Urinary glycosaminoglycan levels • Growth parameters (height, weight, head circumference) • Distance walked in the 6-min walk test • Left ventricular mass index (calculated by echocardiography) • Pulmonary function (measured by forced expiratory volume in 1 s and forced vital capacity) • Liver and spleen size (as estimated by palpation) • Prevalence of cardiac- and pulmonary-related hospitalizations • Deaths (including age at death and cause of death) | |
Secondary endpoints | |
• Evaluation of the natural history of MPS II (based on the following signs and symptoms: hepatosplenomegaly, central nervous system involvement, skeletal involvement, and signs and symptoms of cardiac, pulmonary, and ear, nose, and throat involvement) • Evaluation of dosing of idursulfase (prescribed dose, administered dose, total infusion time, missed infusions, and reasons for missed infusions) • Scoring in five domains in the patient- and parent-reported versions of the HS-FOCUS questionnaire |