Overview

Safety and Tolerability of VGR-R01 in Patients With Bietti Crystalline Dystrophy

Status:
Not yet recruiting
Trial end date:
2024-03-01
Target enrollment:
0
Participant gender:
All
Summary
An Open-Label, Non-Randomized, Uncontrolled, Single-dose Pilot Study of VGR-R01 in Patients with Bietti Crystalline Dystrophy.
Phase:
Early Phase 1
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Beijing Tongren Hospital
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
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 requirements of the study. -

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. 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 of systemic medications that may cause eye injure;

7. Live attenuated vaccines is 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 cessation of anti-platelet agents the platelet
function does not recover;

11. Use of any corticosteroids, other immunosuppressive drug(s) or antipsychotic drugs
(eg. antidepressant, etc.) within 3 months prior to enrollment;

12. Have contraindications for corticosteroids or immunosuppressant;

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 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. -