New stem cell therapy aims to free patients from lifelong blood transfusions

NCT ID NCT04390971

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a new treatment called ET-01 in 3 people with transfusion-dependent beta-thalassemia, a blood disorder that requires regular blood transfusions. The treatment uses the patient's own stem cells, which are modified and then transplanted back. The goal was to see if it is safe and can reduce or eliminate the need for transfusions by helping the body produce healthy red blood cells.

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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

Locations

  • Affiliated Hospital of Zunyi Medical University

    Zunyi, Guizhou, 563000, China

  • Institute of Hematology & Blood Diseases Hospital

    Tianjin, Tianjin Municipality, 300020, China

  • The Affiliated Hospital of Guizhou Medical University

    Guiyang, Guizhou, 550004, China

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