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.

chronic granulomatous disease granulomatous disease, chronic, X-linked

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Boston Children's Hospital

    RECRUITING

    Boston, Massachusetts, 02115, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Columbia University Irving Medical Center, Morgan Stanley Children's Hospital

    RECRUITING

    New York, New York, 10032, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Duke University

    RECRUITING

    Durham, North Carolina, 27710, United States

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Johns Hopkins All Children's Hospital

    RECRUITING

    St. Petersburg, Florida, 33701, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • University College London Hospital

    RECRUITING

    London, NW1 2PG, United Kingdom

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • University of California, Los Angeles

    RECRUITING

    Los Angeles, California, 90095, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

    Contact

  • University of California, San Francisco

    RECRUITING

    San Francisco, California, 94158, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • University of Minnesota

    RECRUITING

    Minneapolis, Minnesota, 55454, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • University of Utah, Primary Children's Hospital

    RECRUITING

    Salt Lake City, Utah, 84113, United States

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••