First-Ever human test of gene therapy for rare, blinding eye disease begins

NCT ID NCT06467344

Summary

This is the first human trial of an experimental gene therapy called ACDN-01 for people with ABCA4-related eye diseases like Stargardt disease. The study will test the safety and early signs of effectiveness of a single injection into the eye. Fifteen adult participants will be followed for up to five years to see if the treatment can slow or stop vision loss.

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: •••••@•••••

Locations

  • Bascom Palmer Eye Institute

    RECRUITING

    Miami, Florida, 33136, United States

  • Casey Eye Institute OHSU

    RECRUITING

    Portland, Oregon, 97239, United States

  • Cincinnati Eye Institute

    RECRUITING

    Cincinnati, Ohio, 45245, United States

  • Massachusetts Eye and Ear

    RECRUITING

    Boston, Massachusetts, 02114, United States

  • Retina Consultants of Texas

    RECRUITING

    Houston, Texas, 77401, United States

  • Retina Foundation of the Southwest

    RECRUITING

    Dallas, Texas, 75382, United States

  • University of Michigan Kellogg Eye Center

    RECRUITING

    Ann Arbor, Michigan, 48105, United States

  • University of San Francisco

    RECRUITING

    San Francisco, California, 94158, United States

  • Vitreo Retinal Associates

    RECRUITING

    Gainesville, Florida, 32607, United States

  • Wilmer Eye Institute at John Hopkins

    RECRUITING

    Baltimore, Maryland, 21218, United States

Conditions

Explore the condition pages connected to this study.