New pill could cut blood transfusions for blood disorders

NCT ID NCT04987489

Completed Disease control Sponsor: Forma Therapeutics, Inc. Source: ClinicalTrials.gov ↗

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.

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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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