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One-Shot eye injection aims to halt rare genetic vision loss

NCT ID NCT07185256

Summary

This early-stage study is testing a one-time gene therapy injection for two rare, inherited eye diseases that cause progressive vision loss: Best vitelliform macular dystrophy (BVMD) and Autosomal-Recessive Bestrophinopathy (ARB). Researchers will give the experimental drug, OPGx-BEST1, to 10 adult participants to first check if it's safe and to find the best dose. The main goal is to see if a single injection into the eye can help control the disease over five years of follow-up.

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

  • Children's Hospital Los Angeles

    RECRUITING

    Los Angeles, California, 90027, United States

    Contact

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

  • Cincinnati Eye Institute

    RECRUITING

    Cincinnati, Ohio, 45242, United States

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

    Contact

  • Retina Foundation of the Southwest

    RECRUITING

    Dallas, Texas, 75231, United States

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

    Contact

  • Vitreo Retinal Associates

    NOT_YET_RECRUITING

    Gainesville, Florida, 34481, United States

    Contact

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

Conditions

Explore the condition pages connected to this study.