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Gene therapy eye injection fails to improve vision in advanced retinitis pigmentosa trial

NCT ID NCT02556736

First seen Jan 12, 2026 · Last updated May 24, 2026 · Updated 20 times

Summary

This early-stage study tested a single injection of RST-001 into the eye for people with advanced retinitis pigmentosa, a genetic condition causing severe vision loss. All 14 participants had very poor vision (hand motion or worse). The main goal was to check safety, and the treatment did not improve vision. The study was completed, but because it did not show benefit, it did not move forward to include people with better eyesight.

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Contacts and locations

Locations

  • Cincinnati Eye Institute- Edgewood /ID# 236713

    Edgewood, Kentucky, 41017-3415, United States

  • Duke Eye Center /ID# 235715

    Durham, North Carolina, 27705, United States

  • Retina Foundation of the Southwest /ID# 235199

    Dallas, Texas, 75231, United States

  • University of California San Francisco - Mission Bay /ID# 235717

    San Francisco, California, 94158, United States

Conditions

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