To minimize bias, during dosing in 111-302 investigators and subjects will continue to be blinded to the subjects’ treatment allocation in 111-301.
Approximately 33 clinical centers worldwide will participate in the study.
Safety monitoring will be conducted for all subjects after the first dose of BMN 111 and over the duration of the study. This will include 30 minutes of observation for all days of dose administration in conjunction with adverse event (AE) documentation/reporting. If the caregiver is unable or unavailable to administer BMN 111, home health care may be provided. For the first 104 weeks, contact by a study staff member to the caregiver will be required every 4 weeks (±10 days) when there are no study visits or contact in the preceding 4 weeks. After Week 104, contact will be made every 8 weeks (±10 days). During these contacts, study staff will ask about dose administration and seek information on AEs and SAEs by specific questioning. Information on all AEs and serious adverse events (SAEs) should be recorded in the subject’s medical record and on the AE eCRF.