Experimental gene therapy targets rare immune disorder in first human test
NCT ID NCT06959771
First seen Jan 04, 2026 · Last updated May 08, 2026 · Updated 17 times
Summary
This study tests a one-time gene therapy for a rare immune disease called CD40L-HyperIgM syndrome, which causes severe infections and autoimmune problems. One male participant will receive his own stem cells and T cells that have been corrected in the lab using a precise editing technique. Before the infusion, he will get chemotherapy to prepare his body. The goal is to see if the treatment is safe and can restore immune function, with follow-up over 15 years.
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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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National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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