New pill could cut blood transfusions for blood disorders
NCT ID NCT04987489
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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A.O.U. Università Studi della Campania "Luigi Vanvitelli"
Naples, 80138, Italy
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AORN A. Cardarelli
Naples, 80131, Italy
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Abu El-Reesh El-Mounira Children University Hospital
Cairo, Egypt, 4241317, Egypt
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Barts Health NHS Trust - The Royal London Hospital
London, E1 1FR, United Kingdom
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CHU Sainte-Justine Mother and Child University Hospital
Montreal, Quebec, H3T 1C5, Canada
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Cairo University
Cairo, 12613, Egypt
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Children's Hospital of Atlanta
Atlanta, Georgia, 30342, United States
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Children's Hosptl Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Children's National Health Hospital
Washington D.C., District of Columbia, 20010, United States
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Chronic Care Center
Baabda, RGWX 4CG, Lebanon
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Duke University
Durham, North Carolina, 27710, United States
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East Carolina University
Greenville, North Carolina, 27858, United States
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Fondazione IRCSS Ca' Granda Ospedale Maggiore Policlinico
Milan, 20122, Italy
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Galliera Hospital Centro Anemie Congenite
Genova, 16128, Italy
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Hammersmith Hospital - London
London, W12 0HS, United Kingdom
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Hospital Nini
Tripoli, 1434, Lebanon
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Kings College London
London, SE5 9RS, United Kingdom
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Manchester University NHS Foundation Trust
Manchester, M13 9WL, United Kingdom
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Master Centre for Canada
Mississauga, Ontario, L4W 4XI, Canada
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Sandwell and West Birmingham NHS Trust SCAT/ haematology
Birmingham, B18 7QH, United Kingdom
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TOI Clinical Research
Cerritos, California, 90703, United States
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The Hospital for Sick Children
Toronto, Ontario, M5G 1X8, Canada
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UCI Health
Orange, California, 92868, United States
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UCSF Oakland Benioff ChildHosp
Oakland, California, 94609, United States
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University College Hospital - University College London Hospitals NHS Foundation Trust
London, NW1 2PG, United Kingdom
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University Health Network - Toronto General Hospital
Toronto, Ontario, M5G 2C4, Canada
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University of Californ LA-UCLA
Los Angeles, California, 90095, United States
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Weill Medical College of Cornell University
New York, New York, 10065, United States
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[Legal] Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
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[Legal] Children's Hospital Medical Center dba Cincinnati Children's
Cincinnati, Ohio, 45229, United States
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[Legal] Children's Hospital of Orange County on behalf of CHOC Children's Hospital of Orange County
Orange, California, 92868, United States
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[Legal] Dr. Vince Clinical Research, LLC and Dr. Vince Clinical Research, P.A.
Detroit, Michigan, 48201-2018, United States