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New pill could boost red blood cells in thalassemia patients

NCT ID NCT04770753

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

What the study statuses mean

This study's is highlighted.

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 26, 2026 · Last updated Aug 28, 2026 · Updated 4 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.

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

194 people

The number who actually took part.

Started

Dec 2021

Expected to finish

Dec 2028

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: * 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 Hb electrophoresis, Hb high-performance liquid chromatography (HPLC)), and/or deoxyribonucleic acid (DNA) analysis; * Hb concentration ≤10.0 grams per deciliter (g/dL) (100.0 grams per liter \[g/L\]), based on an average of at least 2 Hb concentration measurements (separated by ≥7 days) collected during the Screening Period; * Non-transfusion-dependent, defined as ≤5 red blood cell (RBC) units during the 24-week period before randomization; and no RBC transfusions ≤8 weeks before providing informed consent and no RBC transfusions during the Screening Period; * If taking hydroxyurea, the hydroxyurea dose must be stable for ≥16 weeks before randomization; * Women of child-bearing potential (WOCBP) must be abstinent of sexual activities that may result in pregnancy as part of their usual lifestyle or agree to use 2 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 (HbS) or hemoglobin C (HbC); * 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 ≥18 weeks before randomization; * Currently receiving treatment with hematopoietic stimulating agents; the last dose must have been administered ≥18 weeks before randomization; * History of malignancy, (active or treated) ≤5 years before providing informed consent; * History of active and/or uncontrolled cardiac or pulmonary disease ≤6 months before providing informed consent, except for nonmelanomatous skin cancer in situ, cervical carcinoma in situ, or breast carcinoma in situ; * Hepatobiliary disorders; * Estimated glomerular filtration rate \<45 milliliters per minute (mL/min)/1.73 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) ≤16 weeks 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 ≥10 weeks before randomization; * Known allergy to mitapivat or its excipients (microcrystalline cellulose, croscarmellose sodium, sodium stearyl fumarate, mannitol, and magnesium stearate, Opadry® II Blue \[hypromellose, titanium dioxide, lactose monohydrate, triacetin, and FD\&C Blue #2\]); * 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 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)

More trials for these conditions

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