Gene fix trial aims to tame rare immune disease
NCT ID NCT07113743
First seen Jun 27, 2026 · Last updated Aug 14, 2026 · Updated 2 times
Summary
This study tests a gene therapy for people with X-linked chronic granulomatous disease (X-CGD), a genetic condition that weakens the immune system and leads to severe infections. Researchers take the patient's own stem cells, add a working copy of the faulty gene, and return the cells to the patient after mild chemotherapy. The goal is to restore immune function and reduce infection risk. Up to 10 participants aged 3 to 60 will be followed for 2 years, then long-term for 13 more years.
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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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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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