Gene therapy aims to fix immune system in rare disease
NCT ID NCT06876363
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a one-time gene therapy called EN-374 for people with X-linked chronic granulomatous disease (X-CGD), a rare immune disorder that causes severe infections. The treatment uses a modified virus to deliver a working gene to blood stem cells, aiming to restore the ability of white blood cells to kill germs. The study will enroll about 15 males aged 3 months and older, and will check safety and whether the therapy increases functional immune cells.
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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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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
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Columbia University Irving Medical Center, Morgan Stanley Children's Hospital
RECRUITINGNew York, New York, 10032, United States
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Duke University
RECRUITINGDurham, North Carolina, 27710, United States
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Johns Hopkins All Children's Hospital
RECRUITINGSt. Petersburg, Florida, 33701, United States
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University College London Hospital
RECRUITINGLondon, NW1 2PG, United Kingdom
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University of California, Los Angeles
RECRUITINGLos Angeles, California, 90095, United States
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University of California, San Francisco
RECRUITINGSan Francisco, California, 94158, United States
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University of Minnesota
RECRUITINGMinneapolis, Minnesota, 55454, United States
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University of Utah, Primary Children's Hospital
RECRUITINGSalt Lake City, Utah, 84113, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Experimental gene therapy targets rare immune disorder
- Experimental gene therapy aims to fix immune system in rare childhood disease
- Gene therapy hope for rare immune disease fails to launch
- Gene fix trial aims to tame rare immune disease
- Gene-Editing breakthrough aims to fix immune cells in rare disease
- MRNA therapy gives immune cells a temporary boost against rare infection disease