One-Shot gene therapy aims to halt rare, blinding eye disease in young people

NCT ID NCT05956626

First seen Nov 01, 2025 · Last updated Apr 06, 2026 · Updated 21 times

Summary

This study is testing a one-time gene therapy called OCU410ST for Stargardt disease, a genetic condition that causes progressive vision loss in children and young adults. The therapy is delivered via a single injection into the eye and aims to slow or stop the disease by restoring healthy cell function. The trial will enroll 51 participants, with about two-thirds receiving the treatment and the rest being followed as an untreated comparison group.

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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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Contacts and locations

Study contacts

  • Contact

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

  • Contact

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

Locations

  • Advanced Research, LLC

    RECRUITING

    Pompano Beach, Florida, 33064, United States

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

    Contact Email: •••••@•••••

    Contact

  • Associated Retina Consultants

    RECRUITING

    Phoenix, Arizona, 85020, United States

    Contact

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

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

  • Bascom Palmer Eye Institute

    RECRUITING

    Miami, Florida, 33136, United States

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

    Contact

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

  • Boston Children's Hospital

    RECRUITING

    Boston, Massachusetts, 02115, United States

    Contact

    Contact Email: •••••@•••••

  • Duke Eye Center

    RECRUITING

    Durham, North Carolina, 27710, United States

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

    Contact

  • Erie Retina Research, LLC

    RECRUITING

    Erie, Pennsylvania, 16505, United States

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

    Contact

    Contact Email: •••••@•••••

  • Mississippi Retina Associates

    RECRUITING

    Jackson, Mississippi, 39202, United States

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

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

    Contact

  • Retina Consultants of Texas

    RECRUITING

    Bellaire, Texas, 77401, United States

    Contact

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

  • Retina Foundation of the Southwest

    RECRUITING

    Dallas, Texas, 75231, United States

    Contact

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

  • Retina Partners Midwest, P.C.

    RECRUITING

    Carmel, Indiana, 46032, United States

    Contact

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

  • Retinal Consultants Medical Group

    NOT_YET_RECRUITING

    Sacramento, California, 95825, United States

    Contact

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

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

  • The Retina Institute

    RECRUITING

    St Louis, Missouri, 63128, United States

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

    Contact

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

  • Valley Retina Institute

    RECRUITING

    McAllen, Texas, 78503, United States

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

    Contact

  • Vitreo Retinal Associates, P.A.

    RECRUITING

    Gainesville, Florida, 32607, United States

    Contact Email: •••••@•••••

    Contact

Conditions

Explore the condition pages connected to this study.