This is a phase 3 study to evaluate the efficacy and safety of VGR-R01 in subjects with Bietti Crystalline Dystrophy.
This is a multicenter, randomized controlled study which will enroll 45 subjects.
Eligibility
Sex
ALL
Min age
18 Years
Max age
69 Years
Healthy volunteers
No
Key Inclusion Criteria:
1. Able to provide informed consent and comply with requirements of the study;
2. ≥18 years and \<70 years of age;
3. Confirmed diagnosis of Bietti Crystalline Dystrophy and molecular diagnosis of CYP4V2 mutations (homozygotes or compound heterozygotes);
4. Hand Motion ≤ BCVA ≤ 60 ETDRS letters in the study eye;
Key Exclusion Criteria:
1. Have insufficient viable retinal photoreceptor cells based on investigator's decision;
2. Have current ocular or periocular infections, or endophthalmitis;
3. 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;
4. Have intraocular surgery history except cataract surgery in the study eye;
5. Have or potentially require of systemic medications that may cause eye injure;
6. Have contraindications for corticosteroids or immunosuppressant;
7. Unwilling or unable to have the planned follow-up;
8. Abnormal coagulation function or other clinically significant abnormal laboratory results;
9. Have malignancies or history of malignancies;
10. History of immunodeficiency (acquired or congenital); Other protocol defined Inclusion/Exclusion criteria may apply.
Primary outcome measure(s)
Number of subjects with change in BCVA in the study eyes of BCD subjects as assessed by ETDRS — Baseline up to Week 52 Change in the BCVA assessment from screening in the study eye of BCD subjects will be compared to controls
Trial sites (1)
Facility
City
Region
Status
Shanghai Vitalgen Biopharma Co.,Ltd.
Shanghai
Shanghai Municipality
More Shanghai Vitalgen BioPharma Co., Ltd. trials in China
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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