New gene therapy offers hope for 'Bubble Boy' disease patients who failed transplants
NCT ID NCT01306019
Summary
This trial is testing a new gene therapy for patients aged 2 to 50 with X-linked Severe Combined Immunodeficiency (XSCID), a severe genetic immune disorder. The treatment aims to correct the faulty gene in a patient's own blood stem cells and infuse them back after mild chemotherapy. The goal is to restore immune function for patients who have not benefited enough from a previous half-matched bone marrow transplant and lack a perfectly matched donor.
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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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National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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