Can a single injection save sight in a rare retinal disease?
NCT ID NCT06302608
First seen Aug 17, 2026 · Last updated Aug 18, 2026 · Updated 1 time
Summary
This early-stage trial is testing whether a gene therapy called NGGT001, injected under the retina, is safe and tolerable for people with Bietti's crystalline dystrophy, a rare inherited condition that causes progressive vision loss. The study enrolls about 11 adults with a confirmed CYP4V2 gene mutation and reduced vision. Researchers will monitor for side effects and measure changes in visual acuity to see if the treatment might help preserve or improve sight.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- NGGT001, a gene therapy injected under the retina
- What this could lead to
- If successful, this could pave the way for a one-time treatment that slows or stops vision loss in people with Bietti's crystalline dystrophy.
- What could go wrong
- This is a very early, small safety trial, so it is unknown if the therapy will improve vision or how long any benefit might last. Gene therapy injections carry risks like inflammation or retinal damage.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Early phase 1
The earliest testing in people: a first look at safety, in a very small group.
- Participants
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11 people
The number who actually took part.
- Started
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Feb 2023
- Expected to finish
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Nov 2028
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
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 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: 1. Age ≥ 18 years old; 2. Gender unlimited. 3. Diagnosed as crystalline retinal degeneration (BCD). 4. Molecular diagnosis confirms CYP4V2 mutation. 5. The best corrected visual acuity is less than 20/60. 6. Agree to take contraceptive measures within 1 year from the start of the study until administration. 7. Voluntarily sign an informed consent form. Exclusion Criteria: 1. Insufficient number of photoreceptor cells in the retina, such as retinal thickness less than 100 μ m. Or no atrophy or pigmentation in the posterior pole area\<3- Retinal disc. 2. The presence of choroidal neovascularization or other eye lesions caused by BCD, which researchers believe may affect surgical procedures or interfere with the interpretation of clinical endpoints. 3. The use of therapeutic drugs within the first 6 months of enrollment may affect experimental observation, such as Lucentis, Avastin, Conbercept, Triamcinolone acetonide, steroids, etc; 4. The treatment eye has undergone intraocular surgery, such as PDT, vitrectomy, periocular vascular bypass surgery, etc., or requires intraocular surgery during clinical research, such as cataract surgery, retinal laser therapy, etc; 5. Used or may use systemic medications that may cause eye damage, such as psoralen, tamoxifen, etc; 6. Highly sensitive or allergic to the ingredients in the experimental drug (with a history of allergies to two or more drugs or foods); 7. Abnormal and clinically significant physical examination, vital signs, laboratory tests (such as blood routine, urine routine, blood biochemistry, coagulation function, immunological tests, etc.), or abnormal indicators deemed clinically significant by researchers; 8. There are diseases or medical histories that may affect drug safety or internal processes, especially cardiovascular, liver, kidney, endocrine, digestive, lung, neurological, hematological, tumor, immune or metabolic disorders that researchers consider clinically significant. 9. Participated in clinical trials of other drugs or medical devices within 3 months prior to enrollment; 10. Female patients during pregnancy or lactation; 11. Other researchers believe that it is not suitable to participate in the study.
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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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Xiuju Chen
Xiamen, Fujian, 361000, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.