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New oral drug shows promise in reducing blood transfusions for genetic blood disorders

NCT ID NCT04987489

Summary

This Phase 2 clinical trial tested an oral medication called etavopivat in 53 patients with sickle cell disease or thalassemia. The study aimed to see if the drug could safely reduce patients' need for blood transfusions and increase their hemoglobin levels. Researchers measured whether patients could achieve at least a 20% reduction in transfusions or a meaningful increase in hemoglobin over 12 weeks of treatment.

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Contacts and locations

Locations

  • A.O.U. Università Studi della Campania "Luigi Vanvitelli"

    Naples, 80138, Italy

  • AORN A. Cardarelli

    Naples, 80131, Italy

  • Abu El-Reesh El-Mounira Children University Hospital

    Cairo, Egypt, 4241317, Egypt

  • Barts Health NHS Trust - The Royal London Hospital

    London, E1 1FR, United Kingdom

  • CHU Sainte-Justine Mother and Child University Hospital

    Montreal, Quebec, H3T 1C5, Canada

  • Cairo University

    Cairo, 12613, Egypt

  • Children's Hospital of Atlanta

    Atlanta, Georgia, 30342, United States

  • Children's Hosptl Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's National Health Hospital

    Washington D.C., District of Columbia, 20010, United States

  • Chronic Care Center

    Baabda, RGWX 4CG, Lebanon

  • Duke University

    Durham, North Carolina, 27710, United States

  • East Carolina University

    Greenville, North Carolina, 27858, United States

  • Fondazione IRCSS Ca' Granda Ospedale Maggiore Policlinico

    Milan, 20122, Italy

  • Galliera Hospital Centro Anemie Congenite

    Genova, 16128, Italy

  • Hammersmith Hospital - London

    London, W12 0HS, United Kingdom

  • Hospital Nini

    Tripoli, 1434, Lebanon

  • Kings College London

    London, SE5 9RS, United Kingdom

  • Manchester University NHS Foundation Trust

    Manchester, M13 9WL, United Kingdom

  • Master Centre for Canada

    Mississauga, Ontario, L4W 4XI, Canada

  • Sandwell and West Birmingham NHS Trust SCAT/ haematology

    Birmingham, B18 7QH, United Kingdom

  • TOI Clinical Research

    Cerritos, California, 90703, United States

  • The Hospital for Sick Children

    Toronto, Ontario, M5G 1X8, Canada

  • UCI Health

    Orange, California, 92868, United States

  • UCSF Oakland Benioff ChildHosp

    Oakland, California, 94609, United States

  • University College Hospital - University College London Hospitals NHS Foundation Trust

    London, NW1 2PG, United Kingdom

  • University Health Network - Toronto General Hospital

    Toronto, Ontario, M5G 2C4, Canada

  • University of Californ LA-UCLA

    Los Angeles, California, 90095, United States

  • Weill Medical College of Cornell University

    New York, New York, 10065, United States

  • [Legal] Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • [Legal] Children's Hospital Medical Center dba Cincinnati Children's

    Cincinnati, Ohio, 45229, United States

  • [Legal] Children's Hospital of Orange County on behalf of CHOC Children's Hospital of Orange County

    Orange, California, 92868, United States

  • [Legal] Dr. Vince Clinical Research, LLC and Dr. Vince Clinical Research, P.A.

    Detroit, Michigan, 48201-2018, United States

Conditions

Explore the condition pages connected to this study.