Study of the Effect of Etavopivat on Cerebral Hemodynamic Response in Children With Sickle Cell Disease
Status:
Not yet recruiting
Trial end date:
2024-12-15
Target enrollment:
Participant gender:
Summary
Etavopivat (FT-4202) is a potent, selective, orally bioavailable, small-molecule activator of
erythrocyte pyruvate kinase (PKR) being developed by Forma Therapeutics, Inc. and is intended
for use as a treatment for participants with sickle cell disease (SCD) and other inherited
hemoglobinopathies. The clinical hypothesis is that PKR activation will reduce sickle
hemoglobin (HbS) polymerization and improve red blood cell (RBC) membrane function, thereby
reducing RBC sickling and RBC hemolysis that leads to vascular obstruction and anemia, two
hallmarks of SCD pathology. The aim of this study is to determine the effects of etavopivat
on cerebral and muscle hemodynamics