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

Study Overview
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Brief Summary
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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.

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

Official Title
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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

Conditions
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Neovascular Age-related Macular Degeneration

Intervention / Treatment
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* Drug: RRG001


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 50 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Interventional Model Description: dose escalation
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: RRG001 Dose1<br>Frequency of administration: one time injection. | Drug: RRG001<br>* Administered via Subretinal injection. Dosage form: injection solution.<br>|
| Experimental: RRG001 Dose2<br>Frequency of administration: one time injection. | Drug: RRG001<br>* Administered via Subretinal injection. Dosage form: injection solution.<br>|
| Experimental: RRG001 Dose3<br>Frequency of administration: one time injection. | Drug: RRG001<br>* Administered via Subretinal injection. Dosage form: injection solution.<br>|
| Experimental: RRG001 Dose4<br>Frequency of administration: one time injection. | Drug: RRG001<br>* Administered via Subretinal injection. Dosage form: injection solution.<br>|

What is the study measuring?
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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 |