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: 0
• Participant gender: All

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

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Forma Therapeutics, Inc.
• Collaborator: Emory University

Criteria

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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

• 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

• 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

• 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

• Female who is breast feeding or pregnant