← Back to Search

Gene Therapy

RGX-314 Gene Therapy for Wet AMD

Phase 2
Waitlist Available
Research Sponsored by Regenxbio Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Diagnosis of choroidal neovascularization (CNV) secondary to age-related macular degeneration in the study eye previously treated with anti-VEGF
Males or females, aged ≥ 50 years and ≤ 89 years
Must not have
Prior treatment with gene therapy
Active or history of retinal detachment or retinal tear in the study eye
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at week 24 and 48
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing RGX-314, a one-time gene therapy for patients with Wet AMD. It aims to reduce the need for frequent eye injections by using a virus to deliver a helpful gene into the eye. The study will see how well different doses and formulations work over time. RGX-314 is a gene therapy that has shown promise in treating retinal diseases.

Who is the study for?
This trial is for people aged 50-89 with wet age-related macular degeneration (nAMD) who've had some success with anti-VEGF therapy. They must have a certain level of vision in the affected eye and be at least 12 weeks post-cataract surgery. Exclusions include prior gene therapy, recent serious cardiovascular events, or other eye conditions that could affect vision improvement.
What is being tested?
The study tests RGX-314, a one-time gene therapy for nAMD. It aims to see if two different doses from clinical versus commercial formulations are equally effective when delivered subretinally. This phase 2 trial is open label, meaning everyone knows which treatment they're getting.
What are the potential side effects?
While specific side effects aren't listed here, gene therapies like RGX-314 can potentially cause immune reactions, changes in vision, discomfort at the injection site, and inflammation within the eye.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
My eye condition was treated with specific injections due to aging.
Select...
I am between 50 and 89 years old.
Select...
I responded well to previous anti-VEGF therapy.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I have previously undergone gene therapy.
Select...
I have or had a detached retina or tear in my study eye.
Select...
I have scarring or tissue loss in the center of my vision in one eye.
Select...
My vision loss is not due to AMD but another cause.
Select...
My condition is advanced glaucoma in one eye.
Select...
I have not had a heart attack, stroke, or mini-stroke in the last 6 months.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at week 24 and 48
This trial's timeline: 3 weeks for screening, Varies for treatment, and at week 24 and 48 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
Changes in Best Corrected Visual Acuity (BCVA)
Changes in Central Retinal Thickness (CRT)
Incidence and severity of ocular Adverse Events (AEs) and overall AEs
+1 more

Side effects data

From 2021 Phase 1 & 2 trial • 42 Patients • NCT03066258
67%
Retinal Haemorrhage
67%
Intraocular Inflammation
50%
Visual Acuity Reduced/Impairment
33%
Conjunctival Haemorrhage
33%
Photopsia
17%
Pneumonia
17%
Eye Pain
17%
Hypervolaemia
17%
Arthritis
17%
Respiratory Failure
17%
Intraocular Pressure Increased
17%
Vision Blurred
17%
Dry Age-Related Macular Degeneration
17%
Photophobia
100%
80%
60%
40%
20%
0%
Study treatment Arm
Cohort 1
Cohort 4
Cohort 2
Cohort 3
Cohort 5

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

4Treatment groups
Experimental Treatment
Group I: Commercial Formulation Dose 2Experimental Treatment1 Intervention
Dose 2 of RGX-314
Group II: Commercial Formulation Dose 1Experimental Treatment1 Intervention
Dose 1 of RGX-314
Group III: Clinical Formulation Dose 2Experimental Treatment1 Intervention
Dose 2 of RGX-314
Group IV: Clinical Formulation Dose 1Experimental Treatment1 Intervention
Dose 1 of RGX-314
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
RGX-314
2021
Completed Phase 2
~110

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Common treatments for Age-Related Macular Degeneration (AMD), particularly the neovascular (wet) form, include anti-VEGF (vascular endothelial growth factor) drugs and gene therapies like RGX-314. Anti-VEGF drugs work by inhibiting the action of VEGF, a protein that promotes the growth of abnormal blood vessels in the retina, thereby reducing leakage and preventing further vision loss. RGX-314, a gene therapy, aims to provide a long-term solution by delivering a gene that produces a protein to inhibit VEGF, potentially reducing the need for frequent injections. These treatments are vital for AMD patients as they help maintain vision and prevent the progression of retinal damage.

Find a Location

Who is running the clinical trial?

Regenxbio Inc.Lead Sponsor
19 Previous Clinical Trials
2,428 Total Patients Enrolled
REGENXBIO, Inc.Lead Sponsor
19 Previous Clinical Trials
2,428 Total Patients Enrolled
REGENXBIO Inc.Lead Sponsor
24 Previous Clinical Trials
2,685 Total Patients Enrolled

Media Library

RGX-314 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04832724 — Phase 2
Age-Related Macular Degeneration Research Study Groups: Commercial Formulation Dose 1, Clinical Formulation Dose 1, Commercial Formulation Dose 2, Clinical Formulation Dose 2
Age-Related Macular Degeneration Clinical Trial 2023: RGX-314 Highlights & Side Effects. Trial Name: NCT04832724 — Phase 2
RGX-314 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04832724 — Phase 2
~13 spots leftby Oct 2025
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top