Gene therapy trial offers hope for inherited blindness

NCT ID NCT04850118

First seen Apr 22, 2026 · Last updated Apr 23, 2026 · Updated 1 time

Summary

This study tests a gene therapy called AGTC-501 in males aged 12 to 50 with X-linked retinitis pigmentosa, an inherited eye disease that causes vision loss. The treatment aims to improve vision in low light by replacing a faulty gene. Participants receive either a low or high dose of the therapy or no treatment, and their vision is tracked over time.

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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

Locations

  • Bascom Palmer Eye Institute- University of Miami

    Miami, Florida, 33136, United States

  • Baylor Eye Institute

    Houston, Texas, 77030, United States

  • Casey Eye Institute, OHSU

    Portland, Oregon, 97239, United States

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Cincinnati Eye Institute

    Cincinnati, Ohio, 45242, United States

  • Cole Eye Institute - Cleveland Clinic

    Cleveland, Ohio, 44195, United States

  • Duke Eye Center

    Durham, North Carolina, 27710, United States

  • Mayo Clinic

    Rochester, Minnesota, 55905, United States

  • McGill University Health Centre - Glen Site

    Montreal, Quebec, H4A3J1, Canada

  • Mid Atlantic Retina

    Philadelphia, Pennsylvania, 19107, United States

  • Midwest Eye Institute (Retina Partners Midwest)

    Carmel, Indiana, 46290, United States

  • Moorfields Eye Hospital

    London, EC1V 2PD, United Kingdom

  • Ophthalmic Consultants of Boston

    Boston, Massachusetts, 02114, United States

  • Oxford Eye Hospital

    Oxford, OX39DU, United Kingdom

  • Retina Consultants of San Antonio Texas

    San Antonio, Texas, 78240, United States

  • Retina Consultants of Texas

    Bellaire, Texas, 77401, United States

  • Retina Foundation of the Southwest

    Dallas, Texas, 75231, United States

  • Retina Macula Institute of Arizona

    Scottsdale, Arizona, 85255, United States

  • Royal Victorian Eye & Ear Hospital

    East Melbourne, Victoria, 3002, Australia

  • Sydney Eye Hospital

    Sydney, New South Wales, 2000, Australia

  • The Center for Advanced Retinal & Ocular Therapeutics University of Pennsylvania Perelman School of Medicine

    Philadelphia, Pennsylvania, 19104, United States

  • The Retina Clinic London, Institute of Ophthalmology, University College London

    London, W1G7LB, United Kingdom

  • University of Florida Health Jacksonville, Department of Ophthalmology

    Jacksonville, Florida, 32209, United States

  • University of Pittsburgh

    Pittsburgh, Pennsylvania, 15219, United States

  • Wilmer Eye Institute at Johns Hopkins

    Baltimore, Maryland, 21287, United States

Conditions

Explore the condition pages connected to this study.