Patients with significant or suggestive severe CNS vasculopathy (ie, moya moya) of Grade 4 or higher based on MRA read locally. Prior overt stroke (a focal neurological deficit of acute onset) by history or evidence on Screening MRI, history of transient ischemic attack, focal neurological deficit on standardized neurological examination, or concern for moderate or severe neurological deficit (which could be due to stroke) based on a positive "10 questions" screening.Female who is breast feeding or pregnant.Patients, who if female and of childbearing potential, are using acceptable methods of contraception and agree not to donate ova from study start to 90 days after the last dose of study drug, and who if male, are willing to use acceptable methods of contraception and agree not to donate sperm, from study start to 90 days after the last dose of study drug.Hb ≥ 6 grams per deciliter (g/dL) and lesser than or equal to (≤) 9 g/dL at screeningįor participants with aTCD and cTCD and already taking HU, the dose of HU milligram per kilogram (mg/kg) must be stable (no more than a 20% change in dosing except for weight-based changes) for at least 90 days prior to start of study treatment with no anticipated need for dose adjustments except for weight-based changes during the study, in the opinion of the Investigator.Patients in the aTCD cohort must refuse transfusion therapy. This includes patients with cTCD (170-199 centimeter per second ) or aTCD (≥ 200 cm/s). TAMMV greater than or equal to (≥) 170 cm/s in the ICA and/or MCA during the Screening Period and confirmed on 2 occasions and without history of primary ischemic or hemorrhagic stroke, transient ischemic attack, or severe central nervous system (CNS) vasculopathy on magnetic resonance angiography (MRA).SCD genotype may be determined from the results of Hb electrophoresis, high-performance liquid chromatography, or similar testing. Documentation of SCD genotype (HbSS, HbSβ0 -thalassemia) based on prior history of laboratory testing.Type of Participant and Disease Characteristics: Patient's parent, legal guardian, or legal representative has provided documented informed consent and patients have provided age-appropriate assentġ2 to 16 years of age (inclusive) at time of screening At the end of the study, if deemed appropriate by you, your child, and the study doctor, your child may be offered the opportunity to participate in a separate study to continue receiving etavopivat. As part of the study, the participants will be asked to visit the clinic frequently. Each dose should be taken with a glass of water. Etavopivat may be taken with or without food. The dose of 400 mg will be taken as 2 tablets by mouth, each containing 200 mg of etavopivat. The participant will take 400 milligrams (mg) of etavopivat once a day for the 52 weeks. The participant will start a 52-week (1 year) treatment period. The study doctor will determine if the TCD result is conditional or abnormal. In another cohort, participants with conditional TCD or participants with abnormal TCD who are receiving a stable dose of hydroxyurea will be included. In one cohort, participants with conditional transcranial doppler (TCD) or participants with abnormal TCD who are not able to receive hydroxyurea will be included. Participants will be divided into two cohorts depending on their transcranial doppler (TCD) ultrasound results and whether or not they receive hydroxyurea (medication that they may already be taking). The study will test a new medicine, etavopivat, for sickle cell disease and see if it is safe and helpful for participants with sickle cell disease who are at an increased risk of stroke.
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