Could a single stem cell transplant free beta-thalassemia patients from lifelong transfusions?
NCT ID NCT04390971
First seen Feb 11, 2026 · Last updated May 25, 2026 · Updated 10 times
Summary
This early study tested a new stem cell treatment called ET-01 in 3 people with transfusion-dependent beta-thalassemia, a serious blood disorder that requires regular blood transfusions. The treatment uses the patient's own stem cells, which are modified and then given back to help the body produce healthy red blood cells. The main goals were to check safety and see if the transplant could reduce or eliminate the need for transfusions.
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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.
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Contacts and locations
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Locations
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Affiliated Hospital of Zunyi Medical University
Zunyi, Guizhou, 563000, China
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Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality, 300020, China
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The Affiliated Hospital of Guizhou Medical University
Guiyang, Guizhou, 550004, China
Conditions
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