Screening for All Patients. The PI or designee is responsible for administering and obtaining freely given consent, in writing, before the patient enters the study and before any study-related procedures are performed. Each patient will sign an ICF (see Section 10.1.3). A window of up to 60 days is permitted for screening. Screening procedures may be spread over more than one visit. The screening clinic and laboratory procedures listed in Table 1 must be performed and documented. This will include a review of the inclusion and exclusion criteria. The patient’s medical history will be reviewed, and a complete physical examination will be conducted. The medical history must include at least 24 weeks and up to 52 weeks (if available) of RBC transfusion history. A urine drug screen will be performed during screening. The PI, in consultation with the Sponsor, will use professional judgment in allowing the patient to continue participation in the study when evaluating the results of the drug screen, if any positive results are obtained. Iron studies must be performed as early as possible in the Screening Period to identify and initiate iron supplementation, if clinically indicated, in patients with iron deficiency. Patients on iron, folic acid, and/or vitamin B12 supplementation are eligible for the study. If patients are on iron, the dose should be stable for at least 30 days prior to Day 1. All patients must meet all the overall as well as group-specific eligibility requirements listed in Section 5. It will be required that all female patients of childbearing potential have a negative serum pregnancy test to be eligible for the study. If the patient is unable to receive study medication within 60 days of screening, they may be rescreened once. For patients screened more than 60 days prior to the first dose of study medication, all screening assessments must be repeated to confirm eligibility. Patients will also be required to sign a new ICF and should be assigned the same patient number from the initial screening visit. Repeating any screening laboratory test(s) may be permitted on a case-by-case basis with the approval of the Sponsor’s Medical Monitor (or designee). In these instances, repeating a single laboratory test or a subset of the full panel may be acceptable. As part of the screening process, patients will be evaluated to determine whether and which vaccinations are required (see Section 2.3).
