Pill aims to free patients from constant blood transfusions

NCT ID NCT04770779

Summary

This Phase 3 trial tested whether an oral medication called mitapivat could reduce the need for regular blood transfusions in adults with severe forms of thalassemia. 258 participants who required frequent transfusions were randomly assigned to receive either mitapivat or a placebo pill for 48 weeks. The main goal was to see if the drug could cut transfusion needs by at least half, potentially improving quality of life and reducing iron overload complications.

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Contacts and locations

Locations

  • A.O.U Di Modena

    Modena, 41124, Italy

  • A.O.U. San Luigi Gonzaga

    Orbassano, 10043, Italy

  • AOU L. Vanvitelli Universita degli Studi della Campania Luigi Vanvitelli

    Naples, 80138, Italy

  • Acibadem Adana Hospital

    Adana, 1130, Turkey (Türkiye)

  • Akdeniz University Faculty of Medicine

    Antalya, 07059, Turkey (Türkiye)

  • Amsterdam Universitair Medisch Centrum, Locatie AMC

    Amsterdam, 1105 AZ, Netherlands

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • Burjeel Medical City

    Abu Dhabi, United Arab Emirates

  • CHU Hôpital Henri Mondor

    Créteil, 94010, France

  • CHU Hôpital de la Timone

    Marseille, 13385, France

  • Charité - UB - CVK - Medizinische Klinik

    Berlin, 13353, Germany

  • Children's Hospital Agia Sophia, National and Kapodistrian University of Athens Medical School

    Athens, 11527, Greece

  • Children's Hospital Oakland

    Oakland, California, 94609-1809, United States

  • Children's Hospital of Michigan

    Detroit, Michigan, 48201-2196, United States

  • Chronic Care Center

    Beirut, 9999, Lebanon

  • Duke University Medical Center

    Durham, North Carolina, 27710-3038, United States

  • Ege University Faculty of Medicine

    Bornova, 35040, Turkey (Türkiye)

  • Ente Ospedaliero Ospedali Galliera

    Genova, 16128, Italy

  • Erasmus MC

    Westzeedijk 353, 3015 AA, Netherlands

  • Faculty of Medicine Siriraj Hospital

    Bangkok, 10700, Thailand

  • Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

    Milan, 20122, Italy

  • Foothills Medical Centre

    Calgary, Alberta, T2N 2T9, Canada

  • GSH Banco de Sangue de São Paulo

    São Paulo, 04006-002, Brazil

  • Hacettepe University

    Mersin, Turkey (Türkiye)

  • Hospital Ampang

    Pandan Indah, 68000, Malaysia

  • Hospital Das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP

    Ribeirão Preto, 14051-260, Brazil

  • Hospital Pulau Pinang

    Pulau Pinang, 10990, Malaysia

  • Hospital Queen Elizabeth, Kota Kinabalu

    Kota Kinabalu, 88586, Malaysia

  • Hospital Sultanah Aminah Johor Bahru

    Johor Bahru, 80100, Malaysia

  • Hospital Sultanah Bahiyah

    Alor Star, 05460, Malaysia

  • Hospital Tengku Ampuan Afzan

    Kuantan, 25100, Malaysia

  • Hospital Tunku Azizah

    Kuala Lumpur, 50300, Malaysia

  • Hospital Umum Sarawak

    Kuching, 93586, Malaysia

  • Hospital Universitario La Paz

    Madrid, 28046, Spain

  • Hospital Universitario Vall d'Hebron

    Barcelona, 08035, Spain

  • Hospital Universitario Virgen Arrixaca

    Murcia, 30120, Spain

  • Hospital Universitario Virgen del Rocío

    Seville, 41013, Spain

  • Hôpital Edouard Herriot, CHU de Lyon

    Lyon, 69003, France

  • Hôpital Necker Enfants Malades

    Paris, 75015, France

  • Imperial College Healthcare NHS Trust - Hammersmith Hospital

    London, W12 0HS, United Kingdom

  • Ippokrateio General Hospital

    Thessaloniki, 546 42, Greece

  • Istanbul University Faculty of Medicine

    Fatih, 34093, Turkey (Türkiye)

  • King Abdullah International Medical Research Center

    Riyadh, 14611, Saudi Arabia

  • King Chulalongkorn Memorial Hospital

    Pathum Wan, Thailand

  • King Khalid University Hospital

    Riyadh, 90210, Saudi Arabia

  • Laiko General Hospital

    Athens, 115 26, Greece

  • MHAT "Dr. Nikola Vasiliev" AD

    Kyustendil, 2500, Bulgaria

  • Maharaj Nakorn Chiang Mai Hospital

    Chiang Mai, 50200, Thailand

  • Naresuan University Hospital

    Mueang Phitsanulok, 65000, Thailand

  • National Taiwan University Hospital

    Taipei, 100, Taiwan

  • Ospedale "A. Perrino" - Brindisi

    Brindisi, 72100, Italy

  • Ospedale Pediatrico Microcitemico

    Cagliari, 09121, Italy

  • Ospedale Sant'Anna

    Ferrara, 44124, Italy

  • Penn Medicine - University of Pennsylvania Health System

    Philadelphia, Pennsylvania, 19104, United States

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016-7710, United States

  • Phramongkutklao Hospital

    Bangkok, 10400, Thailand

  • Ramathibodi Hospital

    Bangkok, 10400, Thailand

  • Rigshospitalet

    Hovedstaden, 2100, Denmark

  • SHATHD Sofia

    Sofia, 1756, Bulgaria

  • San Diego Hospital, UC San Diego Health

    La Jolla, California, 92093, United States

  • Seattle Cancer Care Alliance, University of Washington

    Seattle, Washington, 98101, United States

  • Srinagarind Hospital, Khon Kaen University

    Khon Kaen, 40000, Thailand

  • Stanford Medicine

    Palo Alto, California, 94304-1601, United States

  • Toronto General Hospital, University Health Network

    Toronto, Ontario, M5G 2C4, Canada

  • UMHAT "Dr. Georgi Stranski" Pleven

    Pleven, 5800, Bulgaria

  • UMHAT "Prof. Dr. Stoyan Kirkovich"

    Stara Zagora, 6000, Bulgaria

  • UMHAT "Sveti Georgi" EAD

    Plovdiv, 4002, Bulgaria

  • Universitair Medisch Centrum Utrecht

    Utrecht, 3584 CX, Netherlands

  • University College London

    London, WC1E 6BT, United Kingdom

  • University General Hospital of Patras

    Achaia, 26504, Greece

  • University Hospital of Ioannina

    Ioannina, 455 00, Greece

  • Universitätsklinikum Essen

    Essen, 45122, Germany

  • Universitätsklinikum Leipzig

    Leipzig, 04103, Germany

  • Weill Cornell Medical Center

    New York, New York, 10065-4870, United States

  • Çukurova University

    Balcalı, 01330, Turkey (Türkiye)

Conditions

Explore the condition pages connected to this study.