Gene therapy aims to restore sight in rare blindness condition
NCT ID NCT07406854
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 3 trial tests a gene therapy called NR082 for Leber's hereditary optic neuropathy (LHON), a genetic condition that causes rapid vision loss. About 95 people aged 12 to 75 with a specific ND4 mutation will receive a single injection of the therapy or a sham procedure. The goal is to see if the treatment can improve eyesight over 52 weeks.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- NR082 (rAAV2-ND4) gene therapy
- What this could lead to
- If successful, this gene therapy could restore some vision in people with LHON caused by ND4 mutations, offering a targeted treatment for this rare inherited blindness.
- What could go wrong
- This is an early-phase trial with only 95 participants, so results may not apply to everyone. Gene therapy carries risks like eye inflammation or no improvement, and long-term effects are unknown.
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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Beijing Tongren Hospital, Capital Medical University
Beijing, Beijing Municipality, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.