One-Time gene shot could free teens from bleeding disorder
NCT ID NCT07080905
First seen Jun 27, 2026 · Last updated Aug 06, 2026 · Updated 4 times
Summary
This study tests a single-dose gene therapy called CSL222 in 20 teenage boys with severe hemophilia B, a bleeding disorder. The goal is to see if the treatment reduces bleeding episodes and improves quality of life. Participants must already be on regular factor IX replacement therapy.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Arthur M. Blank Hospital - Children's Healthcare of Atlanta
RECRUITINGAtlanta, Georgia, 30329, United States
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CHU Hopital de la Timone
RECRUITINGMarseille, 13009, France
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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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Hospital Sant Joan de Déu
RECRUITINGBarcelona, 08950, Spain
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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
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University of Michigan Medical Center
RECRUITINGAnn Arbor, Michigan, 48109, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- A Once-a-Week shot could transform hemophilia Care—Even for those with inhibitors
- Gene Editing's lasting impact: a 10-Year safety watch
- Can a single gene shot free hemophilia b patients from regular infusions?
- Can a video call replace the clinic for hemophilia pain relief?
- Newborn screening study aims to catch rare diseases at birth
- Monthly shot could free kids with hemophilia from frequent infusions