Can a One-Time gene therapy preserve sight for years? a 15-Year study aims to find out
NCT ID NCT03602820
First seen Jul 24, 2026 · Last updated Jul 24, 2026
Summary
This study follows people who previously received a one-time gene therapy injection for a rare inherited eye disease caused by RPE65 mutations. Researchers will test vision and light sensitivity over up to 15 years to see how well the treatment holds up. The goal is to understand the long-term safety and durability of this approach.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- a gene therapy called voretigene neparvovec-rzyl (AAV2-hRPE65v2) given as a one-time injection under the retina
- What this could lead to
- If vision remains stable over many years, this could show that a single gene therapy can provide lasting benefit for people with this rare inherited eye disease.
- What could go wrong
- This is a long-term follow-up study, not a new treatment trial. It cannot prove the therapy works better than other options, and vision may still decline over time.
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.