One-Shot gene therapy could free teens from lifelong bleeding disorder treatments
NCT ID NCT07080905
Summary
This study is testing a single-dose gene therapy called CSL222 in teenage boys with severe hemophilia B. The goal is to see if this one-time treatment can help their bodies produce their own clotting factor, reducing or eliminating the need for regular infusions to prevent bleeds. Researchers will measure how often participants bleed, their quality of life, and monitor for any side effects.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Arthur M. Blank Hospital - Children's Healthcare of Atlanta
RECRUITINGAtlanta, Georgia, 30329, United States
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Chaim Sheba Medical Center
RECRUITINGRamat Gan, 5262000, Israel
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John Radcliffe Hospital - Oxford University Hospitals NHS
RECRUITINGOxford, OX3 9DU, United Kingdom
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St Thomas Hospital
RECRUITINGLondon, SE1 7EH, United Kingdom
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St. Jude Children's Research Hospital
RECRUITINGMemphis, Tennessee, 38105, United States
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University of Florida
RECRUITINGGainesville, Florida, 32611, United States
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University of Michigan Medical Center
RECRUITINGAnn Arbor, Michigan, 48109, United States
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Conditions
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