Early trial tests cell therapy for rare blood disorder
NCT ID NCT04211714
Summary
This early-stage study is testing the safety of an experimental cell therapy called EXG34217 in up to 12 patients with a rare genetic disorder that causes bone marrow failure. The therapy involves collecting a patient's own blood cells, modifying them in a lab to potentially lengthen their telomeres (a key genetic structure), and then infusing them back. The main goal is to see if the treatment is safe and if it can improve patients' blood cell counts.
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Contacts and locations
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Locations
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Cincinnati Children's Hospital
Cincinnati, Ohio, 45229, United States
Conditions
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