Gene therapy trial aims to halt blindness from rare crystal disease
NCT ID NCT05694598
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial tests a gene therapy called VGR-R01 for Bietti Crystalline Dystrophy, a rare inherited eye disease that leads to progressive vision loss. The study involves 12 adults who receive the therapy via injection into the eye. The main goal is to check safety and tolerability, while also measuring any changes in vision.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- VGR-R01 (a gene therapy that delivers a working copy of the CYP4V2 gene using a harmless virus)
- What this could lead to
- If it works, this could point toward a treatment that slows or stops vision loss in people with Bietti Crystalline Dystrophy.
- What could go wrong
- This is a very early, small Phase 1 trial with only 12 people, focused on safety. It may not show clear benefit, and gene therapies can have side effects like inflammation or immune reactions.
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
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
-
About 12 people
The number the study aims to enrol. It can still change while the study runs.
- Started
-
Mar 2023
- Expected to finish
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Sep 2028
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
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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. Able to provide informed consent and comply with requirements of the study; 2. ≥18 years and \<70 years of age; 3. Confirmed diagnosis of Bietti Crystalline Dystrophy and molecular diagnosis of CYP4V2 mutations (homozygotes or compound heterozygotes); 4. BCVA ≤ 60 ETDRS letters in the study 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. Unwilling or unable to have the planned follow-up; 8. Abnormal coagulation function or other clinically significant abnormal laboratory results; 9. Have malignancies or history of malignancies; 10. 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.
Contacts and locations
Locations
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Shanghai Vitalgen Biopharma Co.,Ltd.
Shanghai, Shanghai Municipality, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.