Gene therapy breakthrough offers hope for rare immune disorder
NCT ID NCT06876363
First seen Nov 01, 2025 · Last updated May 15, 2026 · Updated 32 times
Summary
This study tests a new gene therapy called EN-374 for people with X-linked chronic granulomatous disease (X-CGD), a rare immune disorder that causes severe infections. The therapy uses a modified virus to deliver a working gene to fix the immune cells. The trial aims to see if it is safe and can restore the function of infection-fighting neutrophils. It is open to males aged 3 months and older who have had serious infections and lack a suitable bone marrow donor.
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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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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
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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: •••••@•••••
Conditions
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