Gene therapy watch: 5-Year safety check for inherited blindness treatment

NCT ID NCT04312672

First seen Nov 01, 2025 · Last updated May 06, 2026 · Updated 16 times

Summary

This study followed 42 people who had already received a gene therapy for X-linked retinitis pigmentosa, an inherited eye disease that causes vision loss. The main goal was to check the treatment's long-term safety over up to 5 years. Researchers also looked at changes in functional vision, like walking speed in low light and patient-reported vision quality.

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Contacts and locations

Locations

  • Leeds Teaching Hospitals NHS Trust

    Leeds, LS9 7TF, United Kingdom

  • Massachusetts Eye and Ear Infirmary

    Boston, Massachusetts, 02114, United States

  • Moorfields Eye Hospital

    London, EC1V 9EL, United Kingdom

  • Univ of Michigan Medical Center

    Ann Arbor, Michigan, 48105, United States

  • University of Pittsburgh Medical Center

    Pittsburgh, Pennsylvania, 15213, United States

Conditions

Explore the condition pages connected to this study.