Bietti's Crystalline Dystrophy
Conditions
Brief summary
Evaluate the safety and tolerability of NGGT001 subretinal injection for the treatment of crystalline retinal degeneration (BCD)
Interventions
GENETICNGGT001
Safety and Effectiveness of subretinal injection of NGGT001 for treating crystalline retinal degeneration
Sponsors
Xiamen Ophthalmology Center Affiliated to Xiamen University
Study design
Allocation
NON_RANDOMIZED
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. 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence and severity of adverse events (AEs) and serious adverse events (SAEs) | 1 year | Incidence and severity of adverse events (AEs) and serious adverse events (SAEs) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in best-corrected visual acuity | Baseline and Months 1, 2, 3, 4, 5, 6, 9, and 12; the primary efficacy assessment is the change from baseline at Month 12. | 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. |
| Change from baseline in retinal sensitivity assessed by microperimetry | Baseline to Month 12 | 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. |
| Change from baseline in contrast sensitivity | From baseline at Month 12 | 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. |
Countries
China
Contacts
PRINCIPAL_INVESTIGATORLi
Xiamen Ophthalmology Center Affiliated to Xiamen University
Outcome results
None listed