Hypoxic Red Blood Cells in Sickle Cell Anemia

Korábbi kezelés

• Are currently being evaluated for gene therapy;
• Have any clinically significant pulmonary, cardiovascular, endocrine, hepatic, gastrointestinal, renal, infectious, immunological (including significant allo- or auto-immunization) disease, considered not adequately controlled prior to the study;

Más betegség

• Have a diagnosis of HbSC disease, HbSβ+ thalassemia or another SCD variant (excluding HbSS and HbSβ0 thalassemia)
• Have a history of allo-immunization that cannot be managed by the local blood bank;

Laborértékek

• Have received hemoglobin inducers (e.g. erythropoietin) in the 30 days prior to Screening;

Egyéb

• Are not exclusively transfused at the site;
• Are routinely transfused with washed, packed RBC units;
• Are a female of child-bearing potential who is pregnant or planning to become pregnant in the next 14 months;
• Patients who, in the opinion of the Investigator, would not be able or willing to comply with the protocol;
• Is a ward of the state, prisoner, or transient

• Male or female at least 7 years of age;
• Are able to provide informed consent, and assent as applicable, to participate in the study;
• Diagnosis of Sickle Cell Anemia (SCA) (HbSS, HbSβ0 thalassemia) with participation in a chronic transfusion program and have undergone regular transfusions during at least 6 months prior to Screening;
• Have had an average interval of at least 14 days between RBC transfusions over the past 6 months;
• If on iron chelation therapy, have been on a stable dose for ≥3 months prior to screening;