Status:
UNKNOWN
Safety and Tolerability of VGR-R01 in Patients With Bietti Crystalline Dystrophy
Lead Sponsor:
Beijing Tongren Hospital
Conditions:
Bietti Crystalline Dystrophy
Eligibility:
All Genders
18-80 years
Phase:
EARLY_PHASE1
Brief Summary
An Open-Label, Non-Randomized, Uncontrolled, Single-dose Pilot Study of VGR-R01 in Patients with Bietti Crystalline Dystrophy.
Detailed Description
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 w...
Eligibility Criteria
Inclusion
- ≥18 years and \<80 years of age;
- Confirmed diagnosis of Bietti Crystalline Dystrophy;
- Molecular diagnosis of CYP4v2 mutations (homozygotes or compound heterozygotes);
- BCVA ≤ 20/200 in the study eye;
- -8 D \<diopters\< +8 D, 21 mm \< axial lengths ≤ 28 mm in the study eye;
- Normal liver function and renal function；
- Agree to use reliable barrier contraception for 1 year after administration of VGR-R01;
- Able to provide informed consent and comply with requirements of the study. -
Exclusion
- Have insufficient viable retinal photoreceptor cells based on investigator's decision;
- Have current ocular or periocular infections, or endophthalmitis;
- 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;
- Have intraocular surgery history except cataract surgery in the study eye;
- Prior medications which may interfere with the interpretation of study endpoints within six months before screening, eg. anti-VEGF drugs;
- Have or potentially require of systemic medications that may cause eye injure;
- Live attenuated vaccines is expected to be required during the study;
- Participation in a clinical study with an investigational drug or medical device within three months before enrollment;
- History of allergy or sensitivity to investigational drug, medications planned for use in the study;
- Use of anticoagulants, or after 10 days cessation of anti-platelet agents the platelet function does not recover;
- Use of any corticosteroids, other immunosuppressive drug(s) or antipsychotic drugs (eg. antidepressant, etc.) within 3 months prior to enrollment;
- Have contraindications for corticosteroids or immunosuppressant;
- Have complicating systemic diseases that would preclude the planned follow-up;
- Abnormal coagulation function or other clinically significant abnormal laboratory results;
- Have malignancies or history of malignancies;
- History of immunodeficiency (acquired or congenital);
- Females in lactation period;
- Have a history of alcohol or illicit drug addiction;
- Unable or unwilling to comply with the schedule of visits. -
Key Trial Info
Start Date :
September 15 2022
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
May 1 2024
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT05399069
Start Date
September 15 2022
End Date
May 1 2024
Last Update
November 9 2022
Active Locations (1)
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1
Beijing Tongren Hospital
Beijing, Beijing Municipality, China, 100730