This is a multi-centre, single- arm, non-randomized, open-label phase 1/2 clinical trial which enables dosing of the fellow eyes of patients who received VGR-R01 administration in previous studies.
Participants received VGR-R01 administration in the VGR-R01-001 or VGR-R01-101 studies.
Able to provide informed consent and comply with requirements of the study;
Hand Motion ≤ BCVA ≤ 75 ETDRS letters in the second treated eye.
Have insufficient viable retinal photoreceptor cells based on investigator's decision;
Have current ocular or periocular infections, or endophthalmitis;
Have any significant ocular disease/disorder other than BCD, including age-related macular degeneration, diabetic retinopathy, optic neuropathy, significant lens opacity, glaucoma, uveitis, retinal detachment, etc;
Have intraocular surgery history except cataract surgery in the study eye;
Single group
VGR-R01
Biological/VaccineCYP4v2-coding gene delivered by AAV vector
Incidence of adverse events and serious adverse events
Ocular/non-ocular adverse events are collected. The ophthalmic examination will include Best Corrected Visual Acuity (BCVA), Intraocular Pressure (IOP), slit lamp examination, angiography and Optical Coherence Tomography (OCT), etc. If any potential changes accompanied by clinical symptoms, or results in a change of medical intervention, the findings will be considered as clinically significant based on investigator's decision.
Number of participants with clinically significant change from baseline in vital signs, clinically laboratory abnormalities and ophthalmic examination findings
Vital signs (temperature, respiratory rate, pulse rate, systolic and diastolic blood pressure) will be obtained with participant in the seated position, after having sat calmly for at least 10 minutes. Laboratory Tests will include hematology, coagulation, blood chemistry, urinalysis, serology, and pregnancy test, etc. Ophthalmic Examination will include BCVA, IOP, slit lamp examination, angiography and OCT, etc. Clinical significance of the above signs will be determined at the investigator's discretion.
Change from baseline in BCVA
BCVA will be assessed with the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity (VA) chart..
Change from baseline in multi-luminance mobility test (MLMT) score
Subjects will navigate a standardized mobility maze under set conditions as specified times during the study. All light-levels used for testing will be rounded to one of the following specified light levels: 0.1, 1, 4, 10, 50, 125, 250, or 400 lux. The corresponding scores for the above light levels range from 7 to 0, in descending order. -1 point means failing to pass the test at the 400 lux level; the lower the score, the worse the functional vision of the participant.
Change from baseline in optical coherence tomography (OCT)
Change from baseline in central retinal thickness (CRT) as imaged by OCT.
Number of subjects with the presence of immunogenicity
Assessed as presence of systemic cell-mediated or humoral responses to capsid or transgene product.
Visual field index (VFI)
Assessed by Humphery static visual field testing. The VFI is a sophisticated index that range from 0% to 100%. It estimates the percentage of the visual field that the higher the better of visual function. Changes from baseline in VFI will be evaluated.
Central threshold test
The central threshold test is a parameter in the Humphrey visual field test report, used to quantitatively assess the light sensitivity of the fovea (the area that provides the clearest central vision). It is a numerical value expressed in decibels (dB); the higher the value, the better the foveal light sensitivity.
Change from baseline in NEI-VFQ-25 score
As assessed by the National Eye Institute Visual Function Questionnaire -25 (NEI-VFQ-25 questionnaire). NEI-VFQ-25 score ranges from 0 to 100, higher scores indicate better quality of life.
Change from baseline in CLVQOL score
As assessed by the Chinese version of the Low Vision Quality-of-Life Questionnaire (CLVQOL questionnaire). CLVQOL score ranges from 0 to 125; higher scores indicate better quality of life.
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