Bubble boy disease: gene therapy trial offers hope for a cure
NCT ID NCT03311503
First seen Jan 04, 2026 · Last updated May 16, 2026 · Updated 26 times
Summary
This study tests a gene therapy for children under 5 with X-linked severe combined immunodeficiency (SCID-X1), also known as bubble boy disease. Patients receive a low dose of chemotherapy followed by an infusion of their own blood stem cells that have been corrected with a healthy gene. The goal is to restore a working immune system without the need for a donor transplant or lifelong medication.
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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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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: •••••@•••••
Contact
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Cincinnati Children's Hospital Medical Center
RECRUITINGCincinnati, Ohio, 45229, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Emory University/Childrens Healthcare of Atlanta
RECRUITINGAtlanta, Georgia, 30322, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Mattel Children's Hospital - UCLA
RECRUITINGLos Angeles, California, 90095, United States
Contact Phone: •••-•••-••••
Conditions
Explore the condition pages connected to this study.