Could a One-Time gene fix free thalassemia patients from lifelong transfusions?
NCT ID NCT06364774
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a new gene therapy for adults with transfusion-dependent beta-thalassemia, a severe blood disorder. Doctors take the patient's own blood stem cells, add a healthy gene to make normal hemoglobin, and return the cells. The goal is to reduce or stop the need for regular blood transfusions. The study includes 12 participants and is the first human test of this approach.
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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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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.
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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