First-in-Human gene therapy trial aims to free kids from blood transfusions
NCT ID NCT07190001
Summary
This is a very early, small study to test the safety of a single dose of an experimental gene therapy called YOLT-204. It will enroll up to 18 children and teenagers with severe, transfusion-dependent forms of sickle cell disease or beta-thalassemia. The main goals are to see if the treatment is safe and to get a first look at whether it can boost protective 'fetal hemoglobin' levels and reduce the need for blood transfusions.
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Contacts and locations
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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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Guangzhou women and children's medical center
Guangzhou, Guangdong, 510405, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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