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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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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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.
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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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
Contact Phone: •••-•••-•••• Email: •••••@•••••
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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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Contact Phone: •••-•••-•••• Email: •••••@•••••
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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
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••