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Gene Therapy Trial Report

Summary

Safety and Efficacy of RRG001 Gene Therapy in Subjects With Neovascular Age-related Macular Degeneration (nAMD)


NCTID NCT06141460 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Disease Ontology Term DOID:10871
Compound Name RRG001
Sponsor Shanghai Refreshgene Technology Co., Ltd.
Funder Type Industry
Recruitment Status
Enrollment Count 48 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant Anti-VEGF
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Genetic delivery of therapeutic protein
Route of Administration Subretinal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV
Editor Type none
Dose 1 Undisclosed dose 1
Dose 2 Undisclosed dose 2
Dose 3 Undisclosed dose 3
Dose 4 Undisclosed dose 4
Dose 5 Dose range: 1E8 - 1E13 vg/eye

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2023-11-10
Completion Date 2030-12-31
Last Update 2024-11-14

Participation Criteria


Eligible Age >=50 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * Subjects that are willing and able to follow study procedures. * Patinets ≥50 years old. * Clinically diagnosed with CNV secondary to nAMD. * BCVA of the study eye is ≥5 letters and ≤73 letters(Snellen 20/800\~20/40), as well as that of the other eye ≥19 letters(Snellen 20/400). * Responding to anti-VEGF Exclusion Criteria: * Presence of any other eye diseases other than nAMD in study eye that would affect the treatment or confusing the interpretation of the study results , as judged by the investigator. * CNV or macular edema in the study eye secondary to any causes other than AMD. * Uncontrolled glaucoma. * Uncontrolled hypertension despite medication. * Pregnant or lactating women or individuals with reproductive potential who are unwilling to take effective contraception during the study period.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
FDA Designations
Recent Updates

Resources/Links