What side effects are associated with this type of intervention?

Common treatments for Age-Related Macular Degeneration (AMD) similar to RGX-314, such as anti-VEGF therapies (ranibizumab, bevacizumab, and aflibercept), are generally well-tolerated but can have side effects. These may include eye pain, increased intraocular pressure, inflammation, and rare instances of retinal detachment or endophthalmitis (severe eye infection). Patients may also experience transient visual disturbances following the injection. It is important to discuss these potential risks with a healthcare provider to ensure the best treatment plan.

What prior FDA approvals exist?

The FDA has approved several treatments for Age-Related Macular Degeneration (AMD), particularly targeting neovascular (wet) AMD, which involves abnormal blood vessel growth. Notable treatments include anti-VEGF (vascular endothelial growth factor) therapies such as ranibizumab (Lucentis), aflibercept (Eylea), and bevacizumab (Avastin), which inhibit the growth of new blood vessels in the retina. These treatments are administered via intravitreal injections. RGX-314 represents a novel approach as a gene therapy, aiming to provide a one-time treatment to achieve sustained inhibition of VEGF, potentially reducing the need for frequent injections.

What other types of research exist?

Promising novel alternatives to RGX-314 for Age-Related Macular Degeneration (AMD) patients include: 1) Faricimab, a bispecific antibody targeting both VEGF-A and Ang-2, which has shown efficacy in reducing retinal fluid and improving vision. 2) Brolucizumab, a single-chain antibody fragment that inhibits VEGF and has demonstrated longer durability in maintaining vision. 3) Gene therapy options like ADVM-022, an intravitreal gene therapy delivering an anti-VEGF protein, which is currently in clinical trials and shows potential for sustained efficacy with fewer injections.

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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

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My eye condition was treated with specific injections due to aging.

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I am between 50 and 89 years old.

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I responded well to previous anti-VEGF therapy.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have previously undergone gene therapy.

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I have or had a detached retina or tear in my study eye.

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I have scarring or tissue loss in the center of my vision in one eye.

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My vision loss is not due to AMD but another cause.

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My condition is advanced glaucoma in one eye.

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I have not had a heart attack, stroke, or mini-stroke in the last 6 months.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at week 24 and 48

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%

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

0 / 9Eligibility criteria met

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.