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

Phase 1

Recruiting

• Conditions: Neovascular Age-related Macular Degeneration
• Interventions: Drug: RRG001

• Registration Number: NCT06141460
• Lead Sponsor: Shanghai Refreshgene Technology Co., Ltd.

Brief Summary

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

Detailed Description

This study adopts a multicenter, single-arm, open-label phase I/IIa seamless study design and comprises two stages, i.e., phase I 3+3 dose escalation study and a phase IIa dose expansion study. Four dose groups are preset for the phase I dose escalation study; 3-6 subjects will be enrolled for each dose group, and it is planned to enroll 12-24 subjects. The phase IIa dose expansion study will be conducted successively at the doses after safety assessments of dose 2 and/or dose 3 in phase I are completed. Twelve subjects are expected to be enrolled for each dose group, with a total of 24 subjects will be enrolled overall. The sponsor will assess whether or not to adjust the dose(s) and sample size for the exploration study based on the results in the phase I exploration study.

Study Timeline

• Study First Submitted: 2023-11-10
• Study First Submitted QC: 2023-11-15
• Study First Posted: 2023-11-21
• Study Start: 2023-12-12
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-12
• Last Update Posted: 2024-11-14
• Primary Completion: 2026-04-15
• Study Completion: 2030-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 48

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
RRG001 Dose1	RRG001	Frequency of administration: one time injection.
RRG001 Dose2	RRG001	Frequency of administration: one time injection.
RRG001 Dose3	RRG001	Frequency of administration: one time injection.
RRG001 Dose4	RRG001	Frequency of administration: one time injection.

Primary Outcome Measures

Name	Time	Method
Safety and tolerability	Week 52	incidence of AEs and SAEs

Secondary Outcome Measures

Name	Time	Method
Efficacy after RRG001 injection	Week 52	change in central retinal thickness (CRT)

Trial Locations

Locations (1)

Tianjin Medical University Eye Hospital; 🇨🇳 Tianjin, Tianjin, China