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
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Locations
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A.O.U Di Modena
Modena, 41124, Italy
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A.O.U. San Luigi Gonzaga
Orbassano, 10043, Italy
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AOU L. Vanvitelli Universita degli Studi della Campania Luigi Vanvitelli
Naples, 80138, Italy
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Acibadem Adana Hospital
Adana, 1130, Turkey (Türkiye)
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Akdeniz University Faculty of Medicine
Antalya, 07059, Turkey (Türkiye)
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Amsterdam Universitair Medisch Centrum, Locatie AMC
Amsterdam, 1105 AZ, Netherlands
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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Burjeel Medical City
Abu Dhabi, United Arab Emirates
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CHU Hôpital Henri Mondor
Créteil, 94010, France
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CHU Hôpital de la Timone
Marseille, 13385, France
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Charité - UB - CVK - Medizinische Klinik
Berlin, 13353, Germany
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Children's Hospital Agia Sophia, National and Kapodistrian University of Athens Medical School
Athens, 11527, Greece
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Children's Hospital Oakland
Oakland, California, 94609-1809, United States
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Children's Hospital of Michigan
Detroit, Michigan, 48201-2196, United States
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Chronic Care Center
Beirut, 9999, Lebanon
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Duke University Medical Center
Durham, North Carolina, 27710-3038, United States
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Ege University Faculty of Medicine
Bornova, 35040, Turkey (Türkiye)
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Ente Ospedaliero Ospedali Galliera
Genova, 16128, Italy
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Erasmus MC
Westzeedijk 353, 3015 AA, Netherlands
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Faculty of Medicine Siriraj Hospital
Bangkok, 10700, Thailand
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Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
Milan, 20122, Italy
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Foothills Medical Centre
Calgary, Alberta, T2N 2T9, Canada
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GSH Banco de Sangue de São Paulo
São Paulo, 04006-002, Brazil
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Hacettepe University
Mersin, Turkey (Türkiye)
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Hospital Ampang
Pandan Indah, 68000, Malaysia
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Hospital Das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP
Ribeirão Preto, 14051-260, Brazil
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Hospital Pulau Pinang
Pulau Pinang, 10990, Malaysia
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Hospital Queen Elizabeth, Kota Kinabalu
Kota Kinabalu, 88586, Malaysia
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Hospital Sultanah Aminah Johor Bahru
Johor Bahru, 80100, Malaysia
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Hospital Sultanah Bahiyah
Alor Star, 05460, Malaysia
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Hospital Tengku Ampuan Afzan
Kuantan, 25100, Malaysia
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Hospital Tunku Azizah
Kuala Lumpur, 50300, Malaysia
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Hospital Umum Sarawak
Kuching, 93586, Malaysia
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Hospital Universitario La Paz
Madrid, 28046, Spain
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Hospital Universitario Vall d'Hebron
Barcelona, 08035, Spain
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Hospital Universitario Virgen Arrixaca
Murcia, 30120, Spain
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Hospital Universitario Virgen del Rocío
Seville, 41013, Spain
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Hôpital Edouard Herriot, CHU de Lyon
Lyon, 69003, France
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Hôpital Necker Enfants Malades
Paris, 75015, France
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Imperial College Healthcare NHS Trust - Hammersmith Hospital
London, W12 0HS, United Kingdom
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Ippokrateio General Hospital
Thessaloniki, 546 42, Greece
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Istanbul University Faculty of Medicine
Fatih, 34093, Turkey (Türkiye)
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King Abdullah International Medical Research Center
Riyadh, 14611, Saudi Arabia
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King Chulalongkorn Memorial Hospital
Pathum Wan, Thailand
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King Khalid University Hospital
Riyadh, 90210, Saudi Arabia
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Laiko General Hospital
Athens, 115 26, Greece
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MHAT "Dr. Nikola Vasiliev" AD
Kyustendil, 2500, Bulgaria
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Maharaj Nakorn Chiang Mai Hospital
Chiang Mai, 50200, Thailand
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Naresuan University Hospital
Mueang Phitsanulok, 65000, Thailand
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National Taiwan University Hospital
Taipei, 100, Taiwan
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Ospedale "A. Perrino" - Brindisi
Brindisi, 72100, Italy
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Ospedale Pediatrico Microcitemico
Cagliari, 09121, Italy
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Ospedale Sant'Anna
Ferrara, 44124, Italy
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Penn Medicine - University of Pennsylvania Health System
Philadelphia, Pennsylvania, 19104, United States
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Phoenix Children's Hospital
Phoenix, Arizona, 85016-7710, United States
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Phramongkutklao Hospital
Bangkok, 10400, Thailand
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Ramathibodi Hospital
Bangkok, 10400, Thailand
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Rigshospitalet
Hovedstaden, 2100, Denmark
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SHATHD Sofia
Sofia, 1756, Bulgaria
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San Diego Hospital, UC San Diego Health
La Jolla, California, 92093, United States
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Seattle Cancer Care Alliance, University of Washington
Seattle, Washington, 98101, United States
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Srinagarind Hospital, Khon Kaen University
Khon Kaen, 40000, Thailand
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Stanford Medicine
Palo Alto, California, 94304-1601, United States
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Toronto General Hospital, University Health Network
Toronto, Ontario, M5G 2C4, Canada
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UMHAT "Dr. Georgi Stranski" Pleven
Pleven, 5800, Bulgaria
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UMHAT "Prof. Dr. Stoyan Kirkovich"
Stara Zagora, 6000, Bulgaria
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UMHAT "Sveti Georgi" EAD
Plovdiv, 4002, Bulgaria
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Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX, Netherlands
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University College London
London, WC1E 6BT, United Kingdom
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University General Hospital of Patras
Achaia, 26504, Greece
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University Hospital of Ioannina
Ioannina, 455 00, Greece
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Universitätsklinikum Essen
Essen, 45122, Germany
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Universitätsklinikum Leipzig
Leipzig, 04103, Germany
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Weill Cornell Medical Center
New York, New York, 10065-4870, United States
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Çukurova University
Balcalı, 01330, Turkey (Türkiye)
Conditions
Explore the condition pages connected to this study.