New pill could boost red blood cells in thalassemia patients

NCT ID NCT04770753

Ongoing Disease control Sponsor: Agios Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jul 31, 2026 · Updated 2 times

Summary

This phase 3 trial tests whether the drug mitapivat can improve anemia and reduce fatigue in people with alpha- or beta-thalassemia who do not need regular blood transfusions. About 194 participants received either mitapivat or a placebo daily for 24 weeks. The main goal is to see if more people on mitapivat achieve a meaningful rise in hemoglobin levels compared to those on placebo.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Mitapivat
What this could lead to: If it works, mitapivat could become a new oral treatment option to raise hemoglobin levels and reduce fatigue in people with non-transfusion-dependent thalassemia.
What could go wrong: This is a late-stage trial, but results are not yet published. The drug may not work better than placebo, and side effects are possible. It is not a cure and does not eliminate the need for monitoring.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

alpha thalassemia spectrum beta thalassemia beta-thalassemia intermedia hemoglobin H disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

NON-TRANSFUSION-DEPENDENT ALPHA-THALASSEMIA NON-TRANSFUSION-DEPENDENT BETA-THALASSEMIA

Contacts and locations

Locations

A.O.R.N. "A. Cardarelli"

Naples, 80131, Italy
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, 01130, Turkey (Türkiye)
Akdeniz University Faculty of Medicine

Antalya, 07059, Turkey (Türkiye)
Burjeel Medical City

Abu Dhabi, United Arab Emirates
CHU Hôpital Henri Mondor

Créteil, 94010, France
Cambridge University Hospitals NHS Foundation Trust - Addenbrookes Hospital

Cambridge, CAM, CB2 0QQ, United Kingdom
Children's Hospital Agia Sophia, National and Kapodistrian University of Athens Medical School

Athens, 115 27, Greece
China Medical University, Taiwan

Taichung, 40447, Taiwan
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

Rotterdam, 3015 GD, Netherlands
Faculty of Medicine Siriraj Hospital

Bangkok, 10700, Thailand
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy
GSH Banco de Sangue de São Paulo

São Paulo, 04006-002, Brazil
HEMORIO Instituto Nacional de Hematologia

Rio de Janeiro, 20211-030, Brazil
Hacettepe University

Mersin, 06230, Turkey (Türkiye)
Hopital Edouard Herriot, CHU de Lyon

Lyon, 69003, France
Hospital Ampang

Ampang, 68000, Malaysia
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
Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP

Ribeirão Preto, 14051-260, Brazil
Imperial College Healthcare NHS Trust - Hammersmith Hospital

London, W12 OHS, United Kingdom
Instituto do Cancer do Estado de São Paulo, Hospital das Clínicas da Faculdade de Medicina da Universidad de São Paulo

São Paulo, 05403-000, Brazil
Ippokrateio General Hospital

Thessaloniki, 546 42, Greece
Istanbul University Faculty of Medicine

Fatih, 34093, Turkey (Türkiye)
King Abdulaziz Hospital - Al Ahsa

Al Mubarraz, 36428, Saudi Arabia
King Abdullah International Medical Research Center

Riyadh, 14611, Saudi Arabia
King Chulalongkorn Memorial Hospital

Pathum Wan, 10330, Thailand
Laiko General Hospital

Athens, 115 26, Greece
MHAT "Dr. Nikola Vasiliev" AD

Kyustendil, 2500, Bulgaria
Maharaj Nakorn Chiang Mai Hospital

Chiang Mai, 50200, Thailand
Manchester Royal Infirmary, Manchester University NHS Foundation Trust

Manchester, LAN, M13 9WL, United Kingdom
Massachusetts General Hospital

Boston, Massachusetts, 02114-2696, United States
Naresuan University Hospital

Mueang Phitsanulok, 65000, Thailand
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
Phramongkutklao Hospital

Bangkok, 10400, Thailand
Praxis Pesquisa Medica

Santo André, 09090-790, Brazil
Ramathibodi Hospital

Bangkok, 10400, Thailand
Rigshospitalet

Copenhagen, 2100, Denmark
SHATHD Sofia

Sofia, 1756, Bulgaria
San Diego Hospital, UC San Diego Health

La Jolla, California, 92093, United States
Srinagarind Hospital, Khon Kaen University

Khon Kaen, 40002, Thailand
Stanford Medicine

Palo Alto, California, 94304-1601, United States
Thalassemia Centre Dubai

Dubai, 4545, United Arab Emirates
Toronto General Hospital, University Health Network

Toronto, M5G 2C4, Canada
Universidade de Caxias do Sul

Caxias do Sul, 95070-560, Brazil
Universitair Medisch Centrum Utrecht

Utrecht, 3584 CX, Netherlands
University College London

London, NW1 2PG, United Kingdom
University General Hospital of Patras

Rio, 26504, Greece
Weill Cornell Medical Center

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

Balcalı, 01330, Turkey (Türkiye)

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