Can a single injection restore sight in a rare genetic eye disease?
NCT ID NCT06706427
First seen Aug 12, 2026 · Last updated Aug 13, 2026 · Updated 1 time
Summary
This trial tests an experimental gene therapy called NGGT001 for people with Bietti Crystalline Dystrophy, a rare inherited condition that causes progressive vision loss. The therapy is delivered as a single injection under the retina, aiming to replace a faulty gene with a working copy. The study will evaluate the safety and tolerability of the treatment and look for early signs that it can improve or stabilize vision.
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 using a modified virus to deliver a working copy of the CYP4V2 gene into the retina via subretinal injection.
- What this could lead to
- If successful, this could lead to a one-time treatment that slows or stops vision loss in people with Bietti Crystalline Dystrophy, potentially preserving sight.
- What could go wrong
- This is an early-stage trial with only 12 participants, so results may not apply broadly. Risks include potential side effects from the injection or the viral vector, and the therapy may not improve vision.
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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12 people
The number who actually took part.
- Started
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Mar 2024
- Expected to finish
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Sep 2029
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 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.
1. Age ≥ 18 years old. 2. Male or female. 3. Confirmed diagnosis of BCD. 4. Molecular diagnosis confirmed cytochrome P450 family 4 subfamily v member 2 (CYP4V2) mutation. 5. AAV2 neutralizing antibody titer ≤1:5120. 6. 0.05 ≤ Best Corrected Visual Acuity (BCVA) ≤ 0.3. 7. -6.00D ≤ Refractive error ≤ +3.00D. 8. Agree to take contraceptive measures from the start of the study until one year after medication administration. 9. Volunteer to participate in the study and sign informed consent. Exclusion Criteria: 1. There are choroidal neovascularization or other ocular diseases caused by BCD, which are considered to affect the operation or interfere with the interpretation of clinical endpoint. 2. Patients with evidence of neovascularization or suspected neovascularization, and the presence of tubular reflectivity in the neuroepithelial layer as shown by OCT. 3. Those who had used any of the treatment drugs within 6 months before enrollment, such as Lucentis, Avastin, Conbercept, Triamcinolone acetonide, etc. These may affect the experimental observation. 4. The treated eyes have undergone intraocular surgery, such as photodynamic therapy (PDT), vitrectomy, periocular vascular bypass surgery, etc., or need intraocular surgery in the process of clinical research, such as cataract surgery, retinal laser therapy, etc. 5. Have used or may use systemic drugs that may cause eye damage, such as psoralen, tamoxifen, etc. 6. Highly sensitive or allergic to ingredients in the test drug (with allergic history of two or more drugs or food). 7. Physical examination, vital signs, and laboratory examination (such as blood routine, urine routine, blood biochemistry, coagulation function, immunology examination, etc.) are abnormal and clinically significant, or the investigators believe that the abnormal indicators have clinical significance. 8. There are diseases or medical histories that may affect drug safety or in vivo processes, especially cardiovascular, liver, kidney, endocrine, digestive tract, lung, nerve, blood, tumor, immune or metabolic disorders considered by investigators to be of clinical significance. 9. Participated in clinical trials of other drugs or medical devices within three months before enrollment. 10. Female patients who are pregnant or lactating. 11. Any other conditions which lead the investigator to determine the participant is unsuitable for this 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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Southwest Hospital/Southwest Eye Hospital, Third Military Medical University (Army Medical University)
Chongqing, Chongqing Municipality, 400000, China
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Xiamen Eye Center of Xiamen University
Xiamen, Fujian, 361000, China
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