A Phase 1/2 interventional study of VGR-R01 in Bietti Crystalline Dystrophy, sponsored by Shanghai Vitalgen BioPharma Co., Ltd.. Not yet recruiting at 2 sites in China. Open to participants aged 18 Years to 69 Years. Per ClinicalTrials.gov, last updated 2026-05-14.
Sponsored by Shanghai Vitalgen BioPharma Co., Ltd. · Phase 1/2, Interventional, and Treatment
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.
VGR-R01 is a novel Adeno-associated virus (AAV) vector carrying the human Cytochrome P450 Family 4 Subfamily V Member 2 (CYP4V2) coding sequence. This study will evaluate the safety and efficacy of VGR-R01 administered in the contralateral eye (the second treated eye) of subjects enrolled in the VGR-R01-001 and VGR-R01-101 studies, along with assessments of immunogenicity and vector shedding. Additionally, the long-term safety and efficacy of VGR-R01 treatment will be continuously assessed for up to 5 years after the last dose.
Shanghai Vitalgen BioPharma Co., Ltd. is the lead sponsor of 10 studies on the registry; 4 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
Single-dose Subretinal Administration of VGR-R01
Biological: VGR-R01
CYP4v2-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.
Time frame: Up to Year 5
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.
Time frame: Up to Year 5
Change from baseline in BCVA
BCVA will be assessed with the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity (VA) chart..
Time frame: Year 5
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.
Time frame: Year 5
Change from baseline in optical coherence tomography (OCT)
Change from baseline in central retinal thickness (CRT) as imaged by OCT.
Time frame: Year 5
Number of subjects with the presence of immunogenicity
Assessed as presence of systemic cell-mediated or humoral responses to capsid or transgene product.
Time frame: Year 5
Number of subjects with the presence of vector shedding
Assessed as the presence of vector in peripheral blood or collected tear.
Time frame: Year 5
Fixation stability
Number of treated eyes with changes from baseline in fixation stability with MP-3 Microperimetry. Fixation stability is rated into three levels: stable fixation, relatively unstable fixation, and unstable fixation, with fixed standard reporting on the device settings.
Time frame: Year 5
Light sensitivity
Changes from baseline in light sensitivity with MP-3 Microperimetry. The MP-3 has a stimulus intensity range of 0 to 34 decibels (dB).
Time frame: Year 5
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.
Time frame: Year 5
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.
Time frame: Year 5
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.
Time frame: Year 5
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.
Time frame: Year 5
Plan to share: No
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Bietti Crystalline Dystrophy
Shanghai Vitalgen BioPharma Co., Ltd.