Contact SCGE




Gene Therapy Trial Report

Summary

Study to Evaluate the Efficacy and Safety of VGR-R01 Gene Therapy in Patients With Bietti Crystalline Dystrophy


NCTID NCT06699108 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Bietti Crystalline Dystrophy
Disease Ontology Term DOID:0050664
Compound Name VGR-R01
Compound Alias rAAV2/8-hCYP4V2
Sponsor Shanghai Vitalgen BioPharma Co., Ltd.
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 45 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant CYP4V2
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Subretinal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV2/8
Editor Type none
Dose 1 6.0E10 vg/eye
Dose 2 1.2E11 vg/eye (expansion dose)
Dose 3 2.0E11 vg/eye

Study Record Dates


Current Stage Phase3
Submit Date 2024-11-19
Completion Date 2027-06-30
Last Update 2025-12-24

Participation Criteria


Eligible Age 18 Years - 69 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
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. Hand Motion ≤ 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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
FDA Designations Orphan Drug Designation
Recent Updates Phase 3 enrollment complete, BLA submission target 2H 2026

Resources/Links