New eye injection shows promise for rare Blindness-Causing gene mutation

NCT ID NCT06455826

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage study tested the safety of a new drug called VP-001, given as an injection into the eye, for people with a rare genetic eye disease (PRPF31 mutation) that causes vision loss. Six participants aged 12 and older received multiple doses. The main goal was to check for side effects, not to measure vision improvement.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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

Contacts and locations

Locations

  • Baylor College of Medicine- Alkek Eye Center

    Houston, Texas, 77030, United States

  • Oregon Health and Science University - Casey Eye Institute

    Portland, Oregon, 97239, United States

  • Retina Foundation of the Southwest

    Dallas, Texas, 75321, United States

  • University of Florida Health

    Jacksonville, Florida, 32209, United States

  • University of Michigan Kellogg Eye Center

    Ann Arbor, Michigan, 48105, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.