NCT07586306Not yet recruitingPhase 1
A Clinical Study Evaluating Subretinal Injection of VGR-R01 in the Contralateral Eye of Participants With Bietti's Crystalline Dystrophy Who Had Received VGR-R01 Administration
Shanghai Vitalgen BioPharma Co., Ltd.2 sites in 1 country15 target enrollmentStarted: June 1, 2026Last updated:
Trial Snapshot
- Phase
- Phase 1
- Status
- Not yet recruiting
- Enrollment
- 15
- Locations
- 2
Study Overview
Brief Summary
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.
Detailed Description
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to 69 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •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.
Exclusion Criteria
- •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;
- •Have or potentially require of systemic medications that may cause eye injure;
- •Have contraindications for corticosteroids or immunosuppressant;
- •Abnormal coagulation function or other clinically significant abnormal laboratory results;
- •Have malignancies or history of malignancies;
- •History of immunodeficiency (acquired or congenital); Other protocol defined Inclusion/Exclusion criteria may apply.
Investigators
Study Sites (2)
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