Bietti Crystalline Corneoretinal DystrophyPhase 1/2NCT06706427
Safety and Efficacy Study of NGGT001 in Bietti Crystalline Corneoretinal Dystrophy Subjects
A Phase 1/2 study testing NGGT001 in people with Bietti Crystalline Corneoretinal Dystrophy, run by NGGT (Suzhou) Biotechnology Co., Ltd. at 2 sites in China.
Trial summary
The objective of this study is to evaluate the safety, tolerability, and efficacy of subretinal injection of NGGT001 in patients with Bietti Crystalline Corneoretinal Dystrophy (BCD) and to recommend the optimal dosage for future clinical administration. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Bietti Crystalline Corneoretinal 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
- 12 participants
- Where
- 2 sitesChina
- Study dates
- 21 Mar 2024to 26 Sep 2029
- Phase
- Phase 1/2
- 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
None listed.
Who cannot join
- 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.
- Patients with evidence of neovascularization or suspected neovascularization, and the presence of tubular reflectivity in the neuroepithelial layer as shown by OCT.
- 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.
- 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.
- Have used or may use systemic drugs that may cause eye damage, such as psoralen, tamoxifen, etc.
- Highly sensitive or allergic to ingredients in the test drug (with allergic history of two or more drugs or food).
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- 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.
- 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.
- Participated in clinical trials of other drugs or medical devices within three months before enrollment.
- Female patients who are pregnant or lactating.
- Any other conditions which lead the investigator to determine the participant is unsuitable for this study.
Trial locations and contacts
2 sites in 1 country.
China2 sites
Southwest Hospital/Southwest Eye Hospital, Third Military Medical University (Army Medical University)
Chongqing, Chongqing Municipality, 400000
Xiamen Eye Center of Xiamen University
Xiamen, Fujian, 361000
About this study
Official title: A Phase I/II Study for Subretinal Injection of NGGT001 in Patients With Bietti Crystalline Corneoretinal Dystrophy
The objective of this study is to evaluate the safety, tolerability, and efficacy of subretinal injection of NGGT001 in patients with Bietti Crystalline Corneoretinal Dystrophy (BCD) and to recommend the optimal dosage for future clinical administration.
- Being tested
- NGGT001
- Keywords
- BCD, Bietti Crystalline Corneoretinal Dystrophy
- 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.
NGGT001Biological
Using a recombinant adeno-associated virus (AAV) vector to deliver the gene CYP4V2 via subretinal injection for the treatment of crystalline retinal degeneration.
| Study group | Role | What they receive |
|---|---|---|
| NGGT001 | Experimental | Single Arm: This study is a single-arm design in which all participants receive the NGGT001 gene therapy administered via subretinal injection. Participants are divided into three dose-escalation groups to evaluate safety and efficacy. Receives: Biological: NGGT001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events (AEs) from baseline to 52 weeks.
To evaluate the incidence and severity of AEs, including serious AEs (SAEs) of subretinal injection of NGGT001 in patients with BCD.
Measured: 52 weeks
Evaluate the improvement in BCVA compared to baseline at Week 12, 26 and 52.
To evaluate the BCVA in ETDRS test of subretinal injection of NGGT001 from baseline to W12, 26 and 52.
Measured: Week 12, Week 26 and Week 52
Secondary outcomes (4)
Assessment of microperimetry changes in dB compared to baseline at Week 12, 26 and 52.
Retinal sensitivity will be assessed using the MP-3 Type Microperimeter. This will be measured in dB (decibels), and the retinal sensitivity analysis will be conducted based on these readings.
Measured: Week 12, Week 26 and Week 52
Assessment of contrast sensitivity (CS) changes in dB compared to baseline at Week 12, 26 and 52.
Changes in contrast sensitivity (CS) will be evaluated, and the measurements will be analyzed in decibels (dB).
Measured: Week 12, Week 26 and Week 52
Assessment of Optical Coherence Tomography (OCT) retinal thickness changes compared to baseline at Week 12, 26 and 52.
Retinal thickness will be measured using Optical Coherence Tomography (OCT), and the outcome will be expressed in micrometers (µm).
Measured: Week 12, Week 26 and Week 52
Assessment of Multi-Luminance Mobility Test (MLMT) score changes compared to baseline at Week 12, 26 and 52.
Multi-Luminance Mobility Test (MLMT) will assess subjects' daily life quality across different lighting conditions. The results will be categorized by the different light intensity levels, and the aggregate scores will be used to assess mobility performance
Measured: Week 12, Week 26 and Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Nov 2024
- Study start21 Mar 2024
- Primary completion30 Nov 2025
- Study completion26 Sep 2029 (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 Crystalline Corneoretinal 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
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- NGGT (Suzhou) Biotechnology Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06706427
- Sponsor's protocol ID
- NGGT001-P-2301
- Status last verified
- Aug 2026
- First submitted
- 14 Nov 2024
- Last updated
- 12 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Chen X, Yang L, Wang G, Liu Y, Qu G, Jiang L, Liu Y, Li X. Phase I/II Study of NGGT001 Gene Therapy for Bietti Crystalline Dystrophy. Ophthalmol Sci. 2026 May 31;6(8):101269. doi: 10.1016/j.xops.2026.101269. eCollection 2026 Aug.
PubMed 42437119
Study at a glance
From the registry record, last updated by the sponsor on 12 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Study for Subretinal Injection of NGGT001 in Patients With Bietti Crystalline Corneoretinal Dystrophy
- ClinicalTrials.gov ID
- NCT06706427
- Acronym / sponsor ID
- NGGT001-P-2301
- Keywords
- BCD, Bietti Crystalline Corneoretinal Dystrophy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 12 participants (actual)
- Lead sponsor
- NGGT (Suzhou) Biotechnology Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 21 Mar 2024 (actual)
- Primary completion
- 30 Nov 2025 (actual)
- Study completion
- 26 Sep 2029 (estimated)
- First posted
- 26 Nov 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.
The objective of this study is to evaluate the safety, tolerability, and efficacy of subretinal injection of NGGT001 in patients with Bietti Crystalline Corneoretinal Dystrophy (BCD) and to recommend the optimal dosage for future clinical administration.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| NGGT001 | Experimental | Biological: NGGT001Single Arm: This study is a single-arm design in which all participants receive the NGGT001 gene therapy administered via subretinal injection. Participants are divided into three dose-escalation groups to evaluate safety and efficacy. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (0 listed)
Key exclusion criteria (11 listed)
- 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.
- Patients with evidence of neovascularization or suspected neovascularization, and the presence of tubular reflectivity in the neuroepithelial layer as shown by OCT.
- 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.
- 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.
- Have used or may use systemic drugs 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 |
|---|---|---|
| NGGT001Using a recombinant adeno-associated virus (AAV) vector to deliver the gene CYP4V2 via subretinal injection for the treatment of crystalline retinal degeneration. | Biological | NGGT001 |
Primary outcome
Incidence of adverse events (AEs) from baseline to 52 weeks.
Time frame: 52 weeks
To evaluate the incidence and severity of AEs, including serious AEs (SAEs) of subretinal injection of NGGT001 in patients with BCD.
Evaluate the improvement in BCVA compared to baseline at Week 12, 26 and 52.
Time frame: Week 12, Week 26 and Week 52
To evaluate the BCVA in ETDRS test of subretinal injection of NGGT001 from baseline to W12, 26 and 52.
4 secondary outcomes
Assessment of microperimetry changes in dB compared to baseline at Week 12, 26 and 52.
Time frame: Week 12, Week 26 and Week 52
Assessment of contrast sensitivity (CS) changes in dB compared to baseline at Week 12, 26 and 52.
Time frame: Week 12, Week 26 and Week 52
Assessment of Optical Coherence Tomography (OCT) retinal thickness changes compared to baseline at Week 12, 26 and 52.
Time frame: Week 12, Week 26 and Week 52
Assessment of Multi-Luminance Mobility Test (MLMT) score changes compared to baseline at Week 12, 26 and 52.
Time frame: Week 12, Week 26 and Week 52
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 12 Aug 2026.
- Results publication: PubMed 42437119Chen X, Yang L, Wang G, Liu Y, Qu G, Jiang L, Liu Y, Li X. Phase I/II Study of NGGT001 Gene Therapy for Bietti Crystalline Dystrophy. Ophthalmol Sci. 2026 May 31;6(8):101269. doi: 10.1016/j.xops.2026.101269. eCollection 2026 Aug.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing NGGT001 in people with Bietti Crystalline Corneoretinal Dystrophy, run by NGGT (Suzhou) Biotechnology Co., Ltd. at 2 sites 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 0 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 12 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 2 sites 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 of adverse events (AEs) from baseline to 52 weeks. (time frame: 52 weeks). The study lists 2 primary outcomes in all. 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 NCT06706427, 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 12 Aug 2026.