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

Neovascular Age-related Macular Degeneration Clinical Trial

Official title:

A Phase I/IIa, Dose-escalation and Dose-extension Study to Evaluate the Safety and Efficacy of Single Subretinal Injection of RRG001 in Subjects With Neovascular Age-related Macular Degeneration

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06141460
• Other study ID #: RRG001-101
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: December 5, 2023
• Est. completion date: December 31, 2030

Study information

• Verified date: November 2023
• Source: Shanghai Refreshgene Technology Co., Ltd.
• Contact: Jianfei Li, Master
• Phone: 86-021-58938028
• Email: lijf[@]refreshgene.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

RRG001 is a adeno-associated virus (AAV) vector-based gene therapy for subretinal injection. The long-term, stable therapeutic protein after one time injection for nAMD could potentially reduce the treatment burden and maintain vision.RRG001 is designed to reduce the current treatment burden which often results in undertreatment and vision loss in patients with nAMD receiving anti-VEGF therapy in clinical practice.

Description:

This clinical trial is designed to evaluate the safety and preliminary efficacy of single subretinal injection of RRG001 gene therapy in nAMD.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 48
• Est. completion date: December 31, 2030
• Est. primary completion date: December 31, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 50 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Macular Degeneration
• Neovascular Age-related Macular Degeneration
• Wet Macular Degeneration

Intervention

Drug:
• RRG001
Administered via Subretinal injection. Dosage form: injection solution.

Locations

Country	Name	City	State
China	Tianjin Medical University Eye Hospital	Tianjin	Tianjin

Sponsors (1)

Lead Sponsor	Collaborator
Shanghai Refreshgene Technology Co., Ltd.

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety and tolerability	incidence of AEs and SAEs	Week 52
Secondary	Efficacy after RRG001 injection	change in best-corrected visual acuity (BCVA);	Week 52
Secondary	Efficacy after RRG001 injection	change in central retinal thickness (CRT)	Week 52