Gene therapy fails to improve vision in advanced retinitis pigmentosa trial

NCT ID NCT02556736

First seen Jan 12, 2026 · Last updated May 12, 2026 · Updated 17 times

Summary

This early-stage study tested a gene therapy called RST-001 in 14 people with advanced retinitis pigmentosa, a genetic eye disease that causes severe vision loss. All participants had very poor vision at the start (could only see hand motions or less). The main goal was to check safety, and the therapy did not improve vision. The study was completed, but no benefit was shown.

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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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