New gene therapy aims to fix immune system in rare disease
NCT ID NCT06253507
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a new gene therapy for people with chronic granulomatous disease (CGD), a genetic disorder that weakens the immune system and makes them prone to serious infections. The treatment takes the patient's own blood stem cells, adds a missing gene in the lab, and puts them back into the body. Up to 10 people aged 3 and older will be treated and followed for 3 years to see if the therapy helps control the disease.
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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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