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

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

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

Study Overview

• Status: Recruiting
• Conditions: Neovascular Age-related Macular Degeneration
• Intervention / Treatment: Drug: RRG001

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 Type: Interventional
• Enrollment (Estimated): 48
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Xiaorong Li, Master; Phone Number: 8602223346434; Email: xiaorong.li@163.com

Study Locations

• China
  • Tianjin
    • Tianjin, Tianjin, China, 300384
      • Recruiting
      • Tianjin Medical University Eye Hospital
      • Contact: Li Xiaorong, Professor; Phone Number: 8602223346434; Email: xiaorli@163.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: RRG001 Dose1; Frequency of administration: one time injection.	Drug: RRG001; Administered via Subretinal injection. Dosage form: injection solution.
Experimental: RRG001 Dose2; Frequency of administration: one time injection.	Drug: RRG001; Administered via Subretinal injection. Dosage form: injection solution.
Experimental: RRG001 Dose3; Frequency of administration: one time injection.	Drug: RRG001; Administered via Subretinal injection. Dosage form: injection solution.
Experimental: RRG001 Dose4; Frequency of administration: one time injection.	Drug: RRG001; Administered via Subretinal injection. Dosage form: injection solution.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety and tolerability	incidence of AEs and SAEs	Week 52

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Efficacy after RRG001 injection	change in best-corrected visual acuity (BCVA)；	Week 52
Efficacy after RRG001 injection	change in central retinal thickness (CRT)	Week 52

Collaborators and Investigators

• Sponsor: Shanghai Refreshgene Technology Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): December 12, 2023
• Primary Completion (Estimated): April 15, 2026
• Study Completion (Estimated): December 31, 2030

Study Registration Dates

• First Submitted: November 10, 2023
• First Submitted That Met QC Criteria: November 15, 2023
• First Posted (Actual): November 21, 2023

Study Record Updates

• Last Update Posted (Estimated): November 14, 2024
• Last Update Submitted That Met QC Criteria: November 12, 2024
• Last Verified: November 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases; Retinal Diseases; Retinal Degeneration; Macular Degeneration; Wet Macular Degeneration
• Other Study ID Numbers: RRG001-101

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No