Gene therapy fails to improve vision in advanced retinitis pigmentosa trial
NCT ID NCT02556736
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This completed Phase 1/2a trial tested a gene therapy called RST-001 (AGN-151597) in 14 people with advanced retinitis pigmentosa, a genetic eye disease that causes severe vision loss. All participants had very poor vision (hand motion or worse) at the start. The study aimed to check safety and tolerability, but the treatment did not show any improvement in vision. No serious related side effects were reported, but the lack of efficacy means this approach did not help these patients.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- AGN-151597 (gene therapy)
- What this could lead to
- If it had worked, this gene therapy could have pointed toward a treatment to slow vision loss in retinitis pigmentosa.
- What could go wrong
- The trial did not show improvement in vision. It was very early-stage (Phase 1/2a) with only 14 participants, all with severe vision loss, so results may not apply to others.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
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Cincinnati Eye Institute- Edgewood /ID# 236713
Edgewood, Kentucky, 41017-3415, United States
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Duke Eye Center /ID# 235715
Durham, North Carolina, 27705, United States
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Retina Foundation of the Southwest /ID# 235199
Dallas, Texas, 75231, United States
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University of California San Francisco - Mission Bay /ID# 235717
San Francisco, California, 94158, United States