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: Activation of Pyruvate Kinase-R with Etavopivat (FT-4202) Is Well Tolerated, Improves Anemia, and Decreases Intravascular Hemolysis in Patients with Sickle Cell Disease Treated for up to 12 Weeks

Researchers

Presenter

  • R. Clark Clark Brown

Principal Investigators

  • Santosh L. Saraf

  • Kimberly Cruz

  • Modupe Idowu

  • Theodosia A. Kalfa

  • James Geib

  • Sanjeev Forsyth

  • Patricia Schroeder

  • Eric Wu

  • Patrick Kelly

  • Marilyn J. Telen

Medical Centers

  • Children's Healthcare of Atlanta, Atlanta, GA

  • Advanced Pharma, Miami, FL

  • UNIVERSITY OF ILLINOIS AT CHICAGO, Chicago, IL

  • Memorial Hermann-Texas Medical Center, Houston, TX

  • Division of Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH

  • Duke University Medical Center, Durham, NC

Locations

  • United States

Companies

  • Forma Therapeutics,

Study Components

Therapeutic Area

  • Blood/haematological Diseases

  • Pulmonary/Respiratory Diseases

  • Cardiovascular (CVS)

Disease

  • Deep vein thrombosis

  • Acute chest syndrome

  • Sickle cell anemia

  • Thalassemia

  • Venous thrombosis

  • Anemia

Biomarkers

  • Pyruvate

  • Lactate dehydrogenase

  • Stearoyl-CoA desaturase

  • Red Blood Cells

  • Volatile organic compounds

Drug/Treatment

  • N/A

Outcome

  • N/A

Study Design

  • Randomized

  • Safety

  • Placebo Control

  • Pharmacokinetics

  • Pharmacodynamics

  • Open Label

  • Multiple Ascending Dose

  • Cohort

  • Global

  • Unblinded

Phase

  • II/III

Study Id's

  • N/A

Sponsors

  • N/A

Result

  • N/A

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