Gene therapy takes on rare eye disease in Second-Eye trial
NCT ID NCT07586306
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study is for people with Bietti's crystalline dystrophy, a rare genetic eye disease that causes vision loss. They have already received the gene therapy VGR-R01 in one eye. Now, researchers want to test if giving the same treatment to the other eye is safe and can improve vision. About 15 participants will be followed for side effects and changes in eyesight.
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
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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About 15 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Jun 2026
An estimate. Start dates often move.
- Expected to finish
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Jul 2031
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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18 to 69 years
- 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.
Key Inclusion Criteria: 1. Participants received VGR-R01 administration in the VGR-R01-001 or VGR-R01-101 studies. 2. Able to provide informed consent and comply with requirements of the study; 3. Hand Motion ≤ BCVA ≤ 75 ETDRS letters in the second treated eye. Key Exclusion Criteria: 1. Have insufficient viable retinal photoreceptor cells based on investigator's decision; 2. Have current ocular or periocular infections, or endophthalmitis; 3. Have any significant ocular disease/disorder other than BCD, including age-related macular degeneration, diabetic retinopathy, optic neuropathy, significant lens opacity, glaucoma, uveitis, retinal detachment, etc; 4. Have intraocular surgery history except cataract surgery in the study eye; 5. Have or potentially require of systemic medications that may cause eye injure; 6. Have contraindications for corticosteroids or immunosuppressant; 7. Abnormal coagulation function or other clinically significant abnormal laboratory results; 8. Have malignancies or history of malignancies; 9. History of immunodeficiency (acquired or congenital); Other protocol defined Inclusion/Exclusion criteria may apply.
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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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
2 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Beijing Tongren Hospital
Beijing, Beijing Municipality, China
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Shanghai General Hospital (Shanghai First People's Hospital)
Shanghai, Shanghai Municipality, China
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