New gene therapy aims to free patients from sickle cell pain crises
NCT ID NCT07432867
Summary
This study is testing a new gene therapy called DREAM01 for people with severe sickle cell disease who don't have a matched sibling donor for a bone marrow transplant. Doctors take a patient's own blood stem cells, genetically modify them in a lab to produce healthy hemoglobin, and then transplant them back. The goal is to reduce or eliminate the painful blockages in blood vessels (vaso-occlusive crises) and prevent long-term organ damage.
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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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Department of Biotherapy, Necker-Enfants Malades Hospital
RECRUITINGParis, Île-de-France Region, 75015, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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