Bietti crystalline dystrophy
Conditions
Interventions
low dose:Subretinal injection of low dose NGGT001
high dose:Subretinal injection of high dose NGGT001
Sponsors
The First Affiliated Hospital of Army Medical University
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: 1. Aged >= 18 years; 2. No gender limit; 3. Diagnosis of crystalline retinal degeneration (BCD); 4. Molecular diagnosis confirms CYP4V2 mutation; 5. The best corrected visual acuity is less than 20/60; 6. Consent to contraception from the start of the study to 1 year after administration; 7. Voluntarily sign the informed consent form.
Exclusion criteria
Exclusion criteria: 1. Insufficient number of retinal photoreceptor cells, such as retinal thickness less than 100 um, or posterior pole without atrophy or pigmentosatic area < 3-retinal disc; 2. The presence of choroidal neovascular lesions or other ocular lesions caused by BCD, which the investigator believes interferes with surgical procedures or interferes with clinical endpoint interpretation; 3. Those who have used therapeutic drugs within 6 months before enrollment, which may affect the observation of the trial, such as Lucentis, Avastin, Compacept, triamcinolone acetonide, steroids, etc.; 4. The treated eye has undergone intraspherical surgery, such as PDT, vitrectomy, periocular vascular bypass grafting, etc., or intraspherical surgery is required during clinical research, such as cataract surgery, retinal laser treatment, etc.; 5. Have used or may use systemic drugs that may cause eye injury, such as psoralen, tamoxifen, etc.; 6. Highly sensitive or allergic to the ingredients in the investigational drug (history of allergy to two or more drugs or foods); 7. Physical examination, vital signs, laboratory tests (such as blood routine, urine routine, blood biochemistry, coagulation function, immunological examination, etc.) are abnormal and clinically significant, or abnormal indicators that the investigator considers clinically significant; 8. Presence of diseases or medical history that may affect drug safety or internal processes, especially cardiovascular, liver, kidney, endocrine, digestive tract, pulmonary, nervous, hematology, tumor, immune or metabolic disorders that the investigator considers clinically significant; 9. Have participated in clinical trials of other drugs or medical devices within 3 months before enrollment; 10. Female patients who are pregnant or lactating; 11. When other researchers deem it inappropriate to participate in the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| best corrected visual acuity (BCVA);optical coherence tomography (OCT) of macular area;flash-electroretinogram (f-ERG);Activity of Daily Living (ADL);Reading proficiency test;Fundus photography;Full field stimulus threshold test (FST);Microperimetry; | — |
Countries
China
Contacts
Public ContactLiu Yong
Department of Ophthalmology, the First Affiliated Hospital of Army Medical University
Outcome results
None listed