Experimental gene therapy aims to restore sight in rare eye disease
NCT ID NCT06196827
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early study tests a gene therapy called LX101 in 9 people with a rare inherited eye disease caused by RPE65 gene mutations. The therapy delivers a working copy of the gene into the retina via a single injection. The main goals are to check safety and see if it can improve vision or light sensitivity.
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 using a harmless virus to deliver a working RPE65 gene into the retina)
- What this could lead to
- If successful, this could point toward a treatment that slows or partially reverses vision loss in people with this rare genetic eye disease.
- What could go wrong
- This is a very early Phase 1 trial with only 9 people, so safety and effectiveness are not yet proven. The therapy involves eye surgery and may not improve vision for everyone.
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 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
-
9 people
The number who actually took part.
- Started
-
Jul 2022
- Expected to finish
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Dec 2027
An estimate. End dates often move.
- Lead sponsor
-
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
-
6 years and older
- Sex
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Anyone
- Healthy volunteers
-
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.
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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, China
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Shanghai General Hospital
Shanghai, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.