New pill may cut blood transfusions for thalassemia patients

NCT ID NCT04770779

First seen Nov 01, 2025 · Last updated May 05, 2026 · Updated 30 times

Summary

This phase 3 study tests whether mitapivat, an oral drug, can safely reduce the need for blood transfusions in adults with transfusion-dependent alpha- or beta-thalassemia. Participants receive either mitapivat or a placebo for 48 weeks. The main goal is to see if more people on mitapivat achieve at least a 50% reduction in transfused blood units compared to those on placebo.

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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.