Bietti Crystalline Corneoretinal Dystrophy
Conditions
Keywords
BCD, Bietti Crystalline Corneoretinal Dystrophy
Brief 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.
Interventions
BIOLOGICALNGGT001
Using a recombinant adeno-associated virus (AAV) vector to deliver the gene CYP4V2 via subretinal injection for the treatment of crystalline retinal degeneration.
Sponsors
NGGT (Suzhou) Biotechnology Co., Ltd.
Study design
Allocation
NA
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of adverse events (AEs) from baseline to 52 weeks. | 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. | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Assessment of microperimetry changes in dB compared to baseline at Week 12, 26 and 52. | Week 12, Week 26 and Week 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. |
| Assessment of contrast sensitivity (CS) changes in dB compared to baseline at Week 12, 26 and 52. | Week 12, Week 26 and Week 52 | Changes in contrast sensitivity (CS) will be evaluated, and the measurements will be analyzed in decibels (dB). |
| Assessment of Optical Coherence Tomography (OCT) retinal thickness changes compared to baseline at Week 12, 26 and 52. | Week 12, Week 26 and Week 52 | Retinal thickness will be measured using Optical Coherence Tomography (OCT), and the outcome will be expressed in micrometers (µm). |
| Assessment of Multi-Luminance Mobility Test (MLMT) score changes compared to baseline at Week 12, 26 and 52. | Week 12, Week 26 and Week 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 |
Countries
China
Outcome results
None listed