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

Early Phase 1UnknownNCT05399069

Beijing Tongren Hospital3 enrolled

Overview

An Open-Label, Non-Randomized, Uncontrolled, Single-dose Pilot Study of VGR-R01 in Patients with Bietti Crystalline Dystrophy.

VGR-R01 is a novel AAV vector carrying the human CYP4v2 coding sequence. This study is intended to evaluate the safety and tolerability of a single subretinal administration of VGR-R01. All subjects will undergo 365(±7) days of safety observation and will be encouraged to enroll in an extension study to evaluate the long-term safety of VGR-R01 for a total of five years.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Bietti Crystalline Dystrophy

Interventions

VGR-R01DRUG

Gene Replacement Therapy

Eligibility

Sex: ALLMin age: 18 YearsMax age: 80 Years

Medical Language ↔ Plain English

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 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. -

Locations (1)

Beijing Tongren Hospital

Beijing, Beijing Municipality, China

RECRUITING

Outcomes

Primary Outcomes

Incidence of adverse events

An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant administered a product; the event will not need to have a causal relationship with the treatment.