One-Time gene shot could free teens from bleeding disorder
NCT ID NCT07080905
First seen Jan 10, 2026 · Last updated May 25, 2026 · Updated 20 times
Summary
This study tests a single-dose gene therapy called CSL222 in 20 teenage boys (ages 12 to 17) with severe hemophilia B, a bleeding disorder. The treatment aims to help their bodies produce a missing clotting factor, potentially reducing or stopping the need for regular infusions. Researchers will measure how often bleeding episodes occur and how the therapy affects quality of life.
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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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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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Center for Inherited Blood Disorders
RECRUITINGOrange, California, 92868, 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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La Paz University Hospital
RECRUITINGMadrid, 28046, Spain
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Medical University Vienna
RECRUITINGVienna, 1090, Austria
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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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UZ Leuven - Centrum voor Moleculaire en Vasculaire Biologie
RECRUITINGLeuven, 3000, Belgium
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University of Florida
RECRUITINGGainesville, Florida, 32611, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Michigan Medical Center
RECRUITINGAnn Arbor, Michigan, 48109, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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