Bietti's Crystalline DystrophyEarly Phase 1NCT06302608
Safety and Efficacy Study of NGGT001 in Bietti Crystalline Corneoretinal Dystrophy
A Early Phase 1 study testing NGGT001 in people with Bietti's Crystalline Dystrophy, run by Xiamen Ophthalmology Center Affiliated to Xiamen University at 1 site in China.
Trial summary
Evaluate the safety and tolerability of NGGT001 subretinal injection for the treatment of crystalline retinal degeneration (BCD) Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Bietti's Crystalline Dystrophy
- What is being tested?
- NGGT001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 1 siteChina
- Study dates
- 8 Feb 2023to 25 Nov 2028
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥ 18 years old;
- Gender unlimited.
- Diagnosed as crystalline retinal degeneration (BCD).
- Molecular diagnosis confirms CYP4V2 mutation.
- The best corrected visual acuity is less than 20/60.
- Agree to take contraceptive measures within 1 year from the start of the study until administration.
- Voluntarily sign an informed consent form.
Who cannot join
- 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.
- 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.
- 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;
- 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;
- Used or may use systemic medications that may cause eye damage, such as psoralen, tamoxifen, etc;
- Highly sensitive or allergic to the ingredients in the experimental drug (with a history of allergies to two or more drugs or foods);
Show all 11Show fewer
- 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;
- 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.
- Participated in clinical trials of other drugs or medical devices within 3 months prior to enrollment;
- Female patients during pregnancy or lactation;
- Other researchers believe that it is not suitable to participate in the study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Clinical Trial on the Safety and Effectiveness of Subretinal Injection of NGGT001 for Treating Crystalline Retinal Degeneration
Evaluate the safety and tolerability of NGGT001 subretinal injection for the treatment of crystalline retinal degeneration (BCD)
- Conditions
- Bietti's Crystalline Dystrophy
- Being tested
- NGGT001
- Medical subject headings
- Bietti Crystalline Dystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NGGT001Genetic
Safety and Effectiveness of subretinal injection of NGGT001 for treating crystalline retinal degeneration
| Study group | Role | What they receive |
|---|---|---|
| Dose level 1 | Experimental | Dose level 1 will be administered Receives: Genetic: NGGT001 |
| Dose level 2 | Experimental | Dose level 2 will be administered Receives: Genetic: NGGT001 |
| Dose Level 3 | Experimental | Dose level 3 will be administered Receives: Genetic: NGGT001 |
| Dose Level 4 | Experimental | Dose level 4 will be administered Receives: Genetic: NGGT001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs)
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs)
Measured: 1 year
Secondary outcomes (3)
Change from baseline in best-corrected visual acuity
Best-corrected visual acuity will be measured using Early Treatment Diabetic Retinopathy Study visual acuity charts and reported as the ETDRS letter score. Change from baseline will be calculated separately for the treated study eye and the untreated nonstudy eye. A positive change from baseline indicates improvement in visual acuity.
Measured: Baseline and Months 1, 2, 3, 4, 5, 6, 9, and 12; the primary efficacy assessment is the change from baseline at Month 12.
Change from baseline in retinal sensitivity assessed by microperimetry
Retinal sensitivity will be assessed using the MP-3 microperimeter (Nidek) and reported in decibels. Change from baseline in retinal sensitivity will be calculated separately for the treated study eye and the untreated nonstudy eye. A positive change from baseline indicates increased retinal sensitivity.
Measured: Baseline to Month 12
Change from baseline in contrast sensitivity
Contrast sensitivity will be assessed under mesopic conditions using a sine-wave grating-based system (Metrovision) at spatial frequencies of 0.8, 1.6, 3.2, 6.4, 12.8, and 25.6 cycles per degree. Contrast sensitivity will be reported in decibels. Change from baseline will be calculated separately for the treated study eye and the untreated nonstudy eye. A positive change from baseline indicates improvement in contrast sensitivity.
Measured: From baseline at Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Mar 2024
- Study start8 Feb 2023
- Primary completion25 Nov 2024
- Study completion25 Nov 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Bietti's Crystalline DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Xiamen Ophthalmology Center Affiliated to Xiamen University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Li, Principal investigator, Xiamen Ophthalmology Center Affiliated to Xiamen University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06302608
- Sponsor's protocol ID
- NGGT-BCD-P-2203
- Status last verified
- Aug 2026
- First submitted
- 18 Feb 2024
- Last updated
- 17 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Chen X, Liu X, Cui S, Wang G, Liu Y, Qu G, Jiang L, Liu Y, Li X. Safety and Vision Outcomes Following Gene Therapy for Bietti Crystalline Dystrophy: A Nonrandomized Clinical Trial. JAMA Ophthalmol. 2025 Feb 1;143(2):126-133. doi: 10.1001/jamaophthalmol.2024.5619.
PubMed 39786763
Study at a glance
From the registry record, last updated by the sponsor on 17 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Trial on the Safety and Effectiveness of Subretinal Injection of NGGT001 for Treating Crystalline Retinal Degeneration
- ClinicalTrials.gov ID
- NCT06302608
- Acronym / sponsor ID
- NGGT-BCD-P-2203
- Conditions
- Bietti's Crystalline Dystrophy
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Xiamen Ophthalmology Center Affiliated to Xiamen UniversityUniversity, hospital or other
- Study start
- 8 Feb 2023 (actual)
- Primary completion
- 25 Nov 2024 (actual)
- Study completion
- 25 Nov 2028 (estimated)
- First posted
- 12 Mar 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Evaluate the safety and tolerability of NGGT001 subretinal injection for the treatment of crystalline retinal degeneration (BCD)
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Dose level 1 | Experimental | Genetic: NGGT001Dose level 1 will be administered |
| Dose level 2 | Experimental | Genetic: NGGT001Dose level 2 will be administered |
| Dose Level 3 | Experimental | Genetic: NGGT001Dose level 3 will be administered |
| Dose Level 4 | Experimental | Genetic: NGGT001Dose level 4 will be administered |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Age ≥ 18 years old;
- Gender unlimited.
- Diagnosed as crystalline retinal degeneration (BCD).
- Molecular diagnosis confirms CYP4V2 mutation.
- The best corrected visual acuity is less than 20/60.
Key exclusion criteria (11 listed)
- 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.
- 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.
- 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;
- 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;
- Used or may use systemic medications that may cause eye damage, such as psoralen, tamoxifen, etc;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NGGT001Safety and Effectiveness of subretinal injection of NGGT001 for treating crystalline retinal degeneration | Genetic | Dose Level 3, Dose Level 4, Dose level 1, Dose level 2 |
Primary outcome
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs)
Time frame: 1 year
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs)
3 secondary outcomes
Change from baseline in best-corrected visual acuity
Time frame: Baseline and Months 1, 2, 3, 4, 5, 6, 9, and 12; the primary efficacy assessment is the change from baseline at Month 12.
Change from baseline in retinal sensitivity assessed by microperimetry
Time frame: Baseline to Month 12
Change from baseline in contrast sensitivity
Time frame: From baseline at Month 12
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Aug 2026.
- Background: PubMed 39786763Chen X, Liu X, Cui S, Wang G, Liu Y, Qu G, Jiang L, Liu Y, Li X. Safety and Vision Outcomes Following Gene Therapy for Bietti Crystalline Dystrophy: A Nonrandomized Clinical Trial. JAMA Ophthalmol. 2025 Feb 1;143(2):126-133. doi: 10.1001/jamaophthalmol.2024.5619.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: No
Related trials for Bietti's Crystalline Dystrophy
Active, not recruitingPhase 3
Efficacy and Safety of ZVS101e in Patients With Bietti 's Crystalline Dystrophy
Bietti Crystalline Corneoretinal Dystrophy, Bietti Crystalline Dystrophy
Active, not recruitingEarly Phase 1
Safety and Tolerability of ZVS101e in Patients With Bietti 's Crystalline Dystrophy
Bietti's Crystalline Dystrophy
Not yet recruiting
ZVS101e in Patients With Bietti's Crystalline Dystrophy
Bietti's Crystalline Dystrophy, Bietti Crystalline Corneoretinal Dystrophy
Active, not recruitingPhase 1/2
Safety and Efficacy Study of NGGT001 in Bietti Crystalline Corneoretinal Dystrophy Subjects
Bietti Crystalline Corneoretinal Dystrophy
Active, not recruitingPhase 1/2
Safety and Efficacy of ZVS101e in Patients With Bietti 's Crystalline Dystrophy
Bietti's Crystalline Dystrophy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Li principal investigator
Xiamen Ophthalmology Center Affiliated to Xiamen University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing NGGT001 in people with Bietti's Crystalline Dystrophy, run by Xiamen Ophthalmology Center Affiliated to Xiamen University at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 17 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs) (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06302608, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Aug 2026.