Study of the Effect of Etavopivat on Cerebral Hemodynamic Response in Children With Sickle Cell Disease

Korábbi kezelés

• Any one of the following requiring a medical facility visit within 14 days prior to signing the informed consent form:
  • Vaso-occlusive crisis (VOC)
  • Acute chest syndrome (ACS)
  • Splenic sequestration
  • Dactylitis
• Requires chronic transfusion therapy
• Major surgery involving the stomach or small intestine
• Chemotherapy or radiation within the past 2 years
• History of overt clinical stroke within previous 2 years or any history of an intracranial hemorrhage
• Clinically significant bacterial, fungal, parasitic, or viral infection currently receiving or that will require therapy

Más betegség

• Clinically relevant cardiac or pulmonary disease- e.g., congenital heart defect, uncompensated heart failure, or any unstable cardiac condition, arrhythmic heart condition, pulmonary fibrosis, pulmonary hypertension

Egyéb

• Abnormal TCD in the last 12 months
• RBC transfusion within 60 days of screening
• Severe renal dysfunction at the Screening Visit or on chronic dialysis
• Hepatic dysfunction
• Female who is breast feeding or pregnant

• Homozygous hemoglobin SS (HbSS) or hemoglobin S/beta0 thalassemia (HbS/β0 thal)
• Hemoglobin (Hb): Hb ≤ 9.0 g/dL at baseline
• Concomitant hydroxyurea (HU) therapy is allowed if the dose has been stable for at least 3 months with no anticipated need for dose adjustments during the study and no sign of hematological toxicity