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

Sponsored by Shanghai Vitalgen BioPharma Co., Ltd.

About this trial

Last updated 5 months ago

Study ID

VGR-R01-101

Status

Active not recruiting

Type

Interventional

Phase

Phase 1

Placebo

No

Accepting

18 to 69 Years
All Sexes

Trial Timing

Started 3 years ago

What is this trial about?

A Multicenter, Open-Label, Non-Randomized, Uncontrolled Study of VGR-R01 in Patients with Bietti Crystalline Dystrophy.

What are the Participation Requirements?

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. 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.