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Can a single injection save sight in a rare retinal disease?

NCT ID NCT06302608

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

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

Early phase 1

The earliest testing in people: a first look at safety, in a very small group.

Participants

11 people

The number who actually took part.

Started

Feb 2023

Expected to finish

Nov 2028

An estimate. End dates often move.

Lead sponsor

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

18 years and older

Sex

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

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

  • Xiuju Chen

    Xiamen, Fujian, 361000, China

More trials for these conditions

Other studies related to the condition(s) this trial covers.