One-Time gene injection aims to restore sight in rare eye disease
NCT ID NCT07054632
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 3 trial tests a gene therapy called LX101 in 30 people with inherited retinal dystrophy caused by RPE65 mutations. Participants receive a single injection of LX101 into the eye, while a control group gets no treatment. The study measures changes in functional vision and light sensitivity over time.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- LX101 (a gene therapy that delivers a working RPE65 gene to the retina)
- What this could lead to
- If successful, this could provide a one-time treatment to improve vision and light sensitivity in people with this rare inherited eye disease.
- What could go wrong
- This is a small, early-phase 3 trial with only 30 participants, so results may not apply to everyone. Gene therapy carries risks like inflammation or retinal damage, 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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Shanghai General Hospital
Shanghai, China
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Southwest Hospital of AMU
Chongqing, China
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Tianjin Medical University Ophthalmology Hospital
Tianjin, China
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Zhongshan Ophthalmic Center, Sun Yat sen University
Guangzhou, Guangdong, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.