One-Time gene shot could free teens from bleeding disorder
NCT ID NCT07080905
First seen Jan 10, 2026 · Last updated May 16, 2026 · Updated 18 times
Summary
This study tests a single-dose gene therapy called CSL222 in 20 adolescent boys (ages 12-17) with severe hemophilia B. The treatment aims to help the body produce its own clotting factor, reducing or stopping the need for regular infusions. Researchers will track bleeding rates and quality of life over time.
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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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