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.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
-
30 people
The number who actually took part.
- Started
-
Sep 2023
- Expected to finish
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Aug 2029
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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6 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Subject and/or their guardian signing a written informed consent. Diagnosed with biallelic RPE65 mutation-associated inherited retinal dystrophy. Subjects are 6 years of age or older. Visual acuity of ≤ 20/63 or visual field less than 20 degrees in the eye to be injected. Exclusion Criteria: * Prior gene therapy for IRD and other hereditary eye diseases. Pre-existing eye conditions that would interfere with interpretation of study endpoints. Active intraocular or periocular infections in the study eye. Lacking of sufficient surviving retinal cells. Prior ocular surgery within six months. Complicating systemic diseases or clinically significant abnormal baseline laboratory values. Pre-existing systemic diseases that should not discontinue the use of any retinal toxic compounds. Complicating systemic diseases or clinically significant abnormal baseline laboratory values.
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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.