Could a One-Time gene fix free thalassemia patients from lifelong transfusions?
NCT ID NCT06364774
First seen Nov 01, 2025 · Last updated May 24, 2026 · Updated 22 times
Summary
This study tests a new gene therapy for people with a severe blood disorder called beta-thalassemia, who currently need regular blood transfusions. The therapy involves taking the patient's own blood stem cells, adding a healthy gene to help produce normal red blood cells, and returning them to the patient. The goal is to reduce or eliminate the need for transfusions. The study is early-stage (Phase 1/2) and will enroll 12 participants aged 18 to 40.
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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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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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Conditions
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