New gene therapy aims to rebuild immune system in 'Bubble Boy' disease
NCT ID NCT03311503
Summary
This study is testing a one-time gene therapy for young children with SCID-X1, a life-threatening genetic disorder where the immune system doesn't work. Doctors will collect the child's own blood stem cells, fix the faulty gene in a lab, and then infuse the corrected cells back after a low-dose chemotherapy. The main goal is to see if this safely helps the body rebuild a working immune system without needing a donor transplant.
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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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Boston Childrens Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Cincinnati Children's Hospital Medical Center
RECRUITINGCincinnati, Ohio, 45229, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Emory University/Childrens Healthcare of Atlanta
RECRUITINGAtlanta, Georgia, 30322, United States
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Mattel Children's Hospital - UCLA
RECRUITINGLos Angeles, California, 90095, United States
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Conditions
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