Experimental gene injection aims to halt rare childhood blindness

NCT ID NCT05616793

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

Summary

This study tests a gene therapy called OPGx-001 for people with vision loss caused by LCA5 gene mutations. The treatment is a one-time injection under the retina. The goal is to see if it is safe and can slow or stop vision loss. The study involves 22 participants aged 4 and older.

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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

  • Retina Foundation of the Southwest

    RECRUITING

    Dallas, Texas, 75231, United States

  • University of Pennsylvania Perelman School of Medicine

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

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