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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
Drug Product Name VGR-R01
Drug Product 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
Therapeutic Modality Gene transfer
Therapy Route in vivo
Mechanism of Action Functional gene replacement
Route of Administration Subretinal
Drug Product Type Viral vector
Gene Delivery System Type Viral transduction
Vector Type AAV2/8
Dose 1 6.0E10 vg/eye
Dose 2 1.2E11 vg/eye (expansion dose)
Dose 3 2.0E11 vg/eye

Study Record Dates


Current Phase 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