Plain-English translation of NCT04912843 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Phase 3 — Testing in thousands of people, comparing the treatment against what doctors currently use. This is the last big step before approval.
This trial is testing NR082, a gene therapy that is injected directly into the eye to treat Leber's hereditary optic neuropathy (LHON), a rare inherited eye disease that causes progressive vision loss. The medication is designed to work at the genetic level to help restore vision in people who have this specific condition caused by a mutation in the ND4 gene. Researchers will test whether this treatment is safe and whether it can help people see better.
Leber's hereditary optic neuropathy is a serious inherited condition that causes vision loss, and there are currently no approved treatments specifically for this genetic form of the disease. This trial exists to see whether a new gene therapy approach can safely slow or reverse vision loss in affected patients.
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The trial has two main phases. In the first phase, researchers will test different doses of the medication to find the safest and most effective amount. In the second phase, you will receive one injection of the medication into one eye, while your other eye may receive a sham injection (a procedure that looks like an injection but does not deliver medication). You will need to return for multiple follow-up visits over several months so doctors can monitor your eye health and measure any changes in your vision.
AI-generated summary from trial data · Jun 22, 2026 · Not medical advice
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