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Clinical Trial Report

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
Not yet recruiting
Enrollment Count 45
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
Target Tissue/Cell
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
Dose 4
Dose 5

Study Record Dates


Current Stage Phase3
Submit Date 2024-11-19
Completion Date 2027-06-30
Last Update 2024-11-21

Participation Criteria


Eligible Age 18 Years - 69 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
FDA Designations
Recent Updates Phase I/II preliminary results presented at ASGCT 2024

Resources/Links