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New pill may cut transfusions for thalassemia patients

NCT ID NCT04770779

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

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 28, 2026 · Updated 3 times

Summary

This phase 3 study tests whether mitapivat, an oral medication, can safely reduce the number of blood transfusions needed by 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.

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

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

258 people

The number who actually took part.

Started

Nov 2021

Expected to finish

Jun 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Greater than or equal to (≥)18 years of age at the time of providing informed consent; * Documented diagnosis of thalassemia (β-thalassemia with or without α-globin gene mutations, hemoglobin E (HbE)/β-thalassemia, or α-thalassemia/hemoglobin H (HbH) disease) based on deoxyribonucleic acid (DNA) analysis; * Considered transfusion-dependent, defined as 6 to 20 red blood cells (RBC) units transfused and ≤6-week transfusion-free period during the 24-week period before randomization; * If taking hydroxyurea, the hydroxyurea dose must be stable for ≥16 weeks before randomization; * Women of childbearing potential (WOCBP) must be abstinent of sexual activities that may induce pregnancy as part of their usual lifestyle or agree to use two forms of contraception, one of which must be considered highly effective, from the time of providing informed consent, throughout the study, and for 28 days after the last dose of study drug. The second form of contraception can be an acceptable barrier method; * Written informed consent before any study-related procedures are conducted and willing to comply with all study procedures for the duration of the study. Exclusion Criteria: * Pregnant, breastfeeding, or parturient; * Documented history of homozygous or heterozygous sickle hemoglobin (Hb S) or hemoglobin C (Hb C); * Prior exposure to gene therapy or prior bone marrow or stem cell transplantation; * Currently receiving treatment with luspatercept; the last dose must have been administered ≥36 weeks before randomization; * Currently receiving treatment with hematopoietic stimulating agents; the last dose must have been administered ≥36 weeks before randomization; * History of malignancy (active or treated) ≤5 years before providing informed consent, except for nonmelanomatous skin cancer in situ, cervical carcinoma in situ, or breast carcinoma in situ; * History of active and/or uncontrolled cardiac or pulmonary disease ≤6 months before providing informed consent; * Hepatobiliary disorders; * Estimated glomerular filtration rate \<45 milliliters per minute (mL/min)/1.73 meter (m)\^2 by Chronic Kidney Disease Epidemiology Collaboration creatinine equation; * Nonfasting triglycerides \>440 milligrams per deciliter (mg/dL) (5 millimoles per liter \[mmol/L\]); * Active infection requiring systemic antimicrobial therapy at the time of providing informed consent; * Positive test for hepatitis C virus antibody (HCVAb) with evidence of active HCV infection, or positive test for hepatitis B surface antigen (HBsAg); * Positive test for human immunodeficiency virus (HIV)-1 antibody (Ab) or HIV-2 Ab; * History of major surgery (including splenectomy) ≤6 months before providing informed consent and/or a major surgical procedure planned during the study; * Current enrollment or past participation (≤12 weeks before administration of the first dose of study drug or a timeframe equivalent to 5 half-lives of the investigational study drug, whichever is longer) in any other clinical study involving an investigational treatment or device; * Receiving strong CYP3A4/5 inhibitors that have not been stopped for ≥5 days or a timeframe equivalent to 5 half-lives (whichever is longer); or strong CYP3A4 inducers that have not been stopped for ≥4 weeks or a timeframe equivalent to 5 half-lives (whichever is longer), before randomization; * Receiving anabolic steroids that have not been stopped for at least 4 weeks before randomization. Testosterone replacement therapy to treat hypogonadism is allowed. The testosterone dose and preparation must be stable for ≥12 weeks before randomization; * Known allergy, or other contraindication, to mitapivat or its excipients (microcrystalline cellulose, croscarmellose sodium, sodium stearyl fumarate, mannitol, and magnesium stearate, Opadry® II Blue \[hypromellose, titanium dioxide, lactose monohydrate, triacetin, and Federal Food, Drug, and Cosmetic (FD\&C) Blue #2\]); * Any medical, hematological, psychological, or behavioral condition(s) or prior or current therapy that, in the opinion of the Investigator, may confer an unacceptable risk to participating in the study and/or could confound the interpretation of the study data. Also excluded are: * Participants who are institutionalized by regulatory or court order * Participants with any condition(s) that could create undue influence (including but not limited to incarceration, involuntary psychiatric confinement, and financial or familial affiliation with the Investigator or Sponsor).

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Conditions

The condition(s) this trial relates to.

alpha thalassemia spectrum beta-thalassemia major Hb Bart's hydrops fetalis

As listed by the trial registrant

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

Transfusion-dependent alpha-thalassemia Transfusion-dependent beta-thalassemia

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)

More trials for these conditions

Other studies related to the condition(s) this trial covers.