Bietti Crystalline Dystrophy
Conditions
Interventions
Experimental group:Administration of VGR-R01 via subretinal injection, with concomitant use of immunosuppressive medication
Sponsors
Beijing Tongren Eye Center, Beijing Tongren Hospital, Capital Medical University
Eligibility
Sex/Gender
All
Age
18 Years to 79 Years
Inclusion criteria
Inclusion criteria: 1. =18 years and <80 years of age; 2. Confirmed diagnosis of Bietti Crystalline Dystrophy; 3. Molecular diagnosis of CYP4v2 mutations (homozygotes or compound heterozygotes); 4. BCVA = 20/200 in the study eye; 5. -8 D <diopters< +8 D, 21 mm < axial lengths = 28 mm in the study eye; 6. Normal liver function and renal function 7. Agree to use reliable barrier contraception for 1 year after administration of VGR-R01; 8. Able to provide informed consent and comply with the requirements of the study.
Exclusion criteria
Exclusion criteria: 1. Have insufficient viable retinal photoreceptor cells based on the 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 for cataract surgery in the study eye; 5. Prior medications which may interfere with the interpretation of study endpoints within six months before screening, eg. anti-VEGF drugs; 6. Have or potentially require systemic medications that may cause eye injury; 7. Live attenuated vaccines are expected to be required during the study; 8. Participation in a clinical study with an investigational drug or medical device within three months before enrollment; 9. History of allergy or sensitivity to investigational drug, medications planned for use in the study; 10. Use of anticoagulants, or after 10 days of cessation of anti-platelet agents the platelet function does not recover; 11. Use of any corticosteroids, another immunosuppressive drug(s), or antipsychotic drugs (eg. antidepressant, etc.) within 3 months prior to enrollment; 12. Have contraindications for corticosteroids or immunosuppressants; 13. Have complicating systemic diseases that would preclude the planned follow-up; 14. Abnormal coagulation function or other clinically significant abnormal laboratory results; 15. Have malignancies or a history of malignancies; 16. History of immunodeficiency (acquired or congenital); 17. Females in lactation period; 18. Have a history of alcohol or illicit drug addiction; 19. Unable or unwilling to comply with the schedule of visits.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The safety of The Gene Replacement Therapy by VGR-R01 [ Time Frame: Baseline up to Day 365].; | — |
Secondary
| Measure | Time frame |
|---|---|
| The efficacy of The Gene Replacement Therapy by VGR-R01 [ Time Frame: Baseline up to Day 365].;Changes of functional indexes, behavioral indexes and morphological indexes; | — |
Countries
China
Contacts
Public ContactWenbing Wei
Outcome results
None listed