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New pill could cut blood transfusions for blood disorders

NCT ID NCT04987489

Disease control Sponsor: Forma Therapeutics, 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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026

Summary

This Phase 2 study tested an experimental drug called etavopivat in 53 people with thalassemia or sickle cell disease. The goal was to see if the pill could safely reduce the number of red blood cell transfusions needed and raise hemoglobin levels. Participants took 400 mg of etavopivat once daily, and researchers measured transfusion reduction and hemoglobin response over 12 weeks.

What this could mean

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

Active substance
etavopivat (FT-4202) tablets
What this could lead to
If successful, etavopivat could offer a new oral option to reduce the need for frequent blood transfusions and improve hemoglobin levels in people with thalassemia or sickle cell disease.
What could go wrong
This is an early-phase, small trial (53 participants) with no placebo group, so results may not be definitive. The drug may not work for everyone or could have side effects not yet fully understood.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

53 people

The number who actually took part.

Started

Mar 2022

Finished

Sep 2025

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

12 to 65 years

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: * Provision of consent * Female patients of childbearing potential must use acceptable methods of contraception, male patients are willing to use barrier methods of contraception Cohort A (Sickle Cell Disease Transfusion Cohort) * Confirmed diagnosis of sickle cell disease * Chronically red blood cell transfused (sample or exchange \[manual or via electrophoresis\]) for primary stroke prevention or due to previous stroke. Chronic red blood cell transfusion is defined as: ≥ 6 red blood cell units in the previous 24 weeks before the first dose of study treatment and no transfusion-free period for \> 35 days during that period * At least 24 months of chronic monthly red blood cell transfusions for secondary stroke prevention/treatment of primary stroke (initial completed overt clinical stroke with documented infarction on brain computed tomography \[CT\] or magnetic resonance imaging \[MRI\]) * Prior to screening OR at least 12 months of chronic RBC transfusions for primary stroke prevention (abnormal TCD) prior to screening * Documented adequate monthly transfusions with average HbS ≤ 45% (the upper limit of the established academic community standard) for the previous 12 weeks of red blood cell transfusions before the first dose of study treatment Cohort B (Thalassemia Transfusion Cohort) * Documented diagnosis of β-thalassemia, Hemoglobin E/ β-thalassemia or Hemoglobin H (α-thalassemia), or other thalassemia variant * Chronically transfused, defined as: ≥ 6 red blood cell units in the previous 24 weeks before the first dose of study treatment and no transfusion-free period for \> 35 days during that period Cohort C (Thalassemia Non-transfused Cohort) * Documented diagnosis of β-thalassemia, Hemoglobin E/ β-thalassemia or Hemoglobin H (α-thalassemia), or other thalassemia variant * Hemoglobin ≤ 10 g/dL Exclusion Criteria: * Female who is breast feeding or pregnant * Hepatic dysfunction characterized by: * Alanine aminotransferase (ALT) \> 4.0 × upper limit of normal (ULN) * Direct bilirubin \> 3.0 × ULN * History of cirrhosis * Known human immunodeficiency virus (HIV) positivity * Active hepatitis B or hepatitis C infection * Severe renal dysfunction or on chronic dialysis * History of malignancy within the past 2 years prior to treatment Day 1 requiring systemic chemotherapy and/or radiation. * Patients with malignancy considered surgically cured are eligible (eg, non- melanoma skin cancer, cancer of the cervix in-situ, ductal carcinoma in situ \[Stage 1\], Grade 1 endometrial cancer) * History of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to consent including but not limited to the following: * Unstable angina pectoris or myocardial infarction or elective coronary intervention * Congestive heart failure requiring hospitalization * Uncontrolled clinically significant arrhythmias * Symptomatic pulmonary hypertension

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Conditions

The condition(s) this trial relates to.

alpha thalassemia spectrum anemia beta thalassemia hematologic disorder hemoglobin E-beta-thalassemia syndrome hemoglobin H disease Hemolysis hemolytic anemia hereditary disease inherited hemoglobinopathy sickle cell disease Sickle Cell Trait thalassemia Vaso-Occlusive Crises

As listed by the trial registrant

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

Sickle cell disease Thalassemia

Contacts and locations

Locations

  • A.O.U. Università Studi della Campania "Luigi Vanvitelli"

    Naples, 80138, Italy

  • AORN A. Cardarelli

    Naples, 80131, Italy

  • Abu El-Reesh El-Mounira Children University Hospital

    Cairo, Egypt, 4241317, Egypt

  • Barts Health NHS Trust - The Royal London Hospital

    London, E1 1FR, United Kingdom

  • CHU Sainte-Justine Mother and Child University Hospital

    Montreal, Quebec, H3T 1C5, Canada

  • Cairo University

    Cairo, 12613, Egypt

  • Children's Hospital of Atlanta

    Atlanta, Georgia, 30342, United States

  • Children's Hosptl Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's National Health Hospital

    Washington D.C., District of Columbia, 20010, United States

  • Chronic Care Center

    Baabda, RGWX 4CG, Lebanon

  • Duke University

    Durham, North Carolina, 27710, United States

  • East Carolina University

    Greenville, North Carolina, 27858, United States

  • Fondazione IRCSS Ca' Granda Ospedale Maggiore Policlinico

    Milan, 20122, Italy

  • Galliera Hospital Centro Anemie Congenite

    Genova, 16128, Italy

  • Hammersmith Hospital - London

    London, W12 0HS, United Kingdom

  • Hospital Nini

    Tripoli, 1434, Lebanon

  • Kings College London

    London, SE5 9RS, United Kingdom

  • Manchester University NHS Foundation Trust

    Manchester, M13 9WL, United Kingdom

  • Master Centre for Canada

    Mississauga, Ontario, L4W 4XI, Canada

  • Sandwell and West Birmingham NHS Trust SCAT/ haematology

    Birmingham, B18 7QH, United Kingdom

  • TOI Clinical Research

    Cerritos, California, 90703, United States

  • The Hospital for Sick Children

    Toronto, Ontario, M5G 1X8, Canada

  • UCI Health

    Orange, California, 92868, United States

  • UCSF Oakland Benioff ChildHosp

    Oakland, California, 94609, United States

  • University College Hospital - University College London Hospitals NHS Foundation Trust

    London, NW1 2PG, United Kingdom

  • University Health Network - Toronto General Hospital

    Toronto, Ontario, M5G 2C4, Canada

  • University of Californ LA-UCLA

    Los Angeles, California, 90095, United States

  • Weill Medical College of Cornell University

    New York, New York, 10065, United States

  • [Legal] Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • [Legal] Children's Hospital Medical Center dba Cincinnati Children's

    Cincinnati, Ohio, 45229, United States

  • [Legal] Children's Hospital of Orange County on behalf of CHOC Children's Hospital of Orange County

    Orange, California, 92868, United States

  • [Legal] Dr. Vince Clinical Research, LLC and Dr. Vince Clinical Research, P.A.

    Detroit, Michigan, 48201-2018, United States

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