What is the mechanism of action of this treatment?

The most common treatments for Age-Related Macular Degeneration (AMD) include anti-VEGF therapies and gene therapies like RGX-314. Anti-VEGF treatments work by inhibiting the vascular endothelial growth factor, which reduces abnormal blood vessel growth and leakage in the retina, a hallmark of AMD. RGX-314, a gene therapy administered subretinally, aims to provide a long-term solution by enabling retinal cells to produce a therapeutic protein that inhibits VEGF. These treatments are crucial for AMD patients as they help preserve vision and prevent further retinal damage, thereby improving quality of life.

What side effects are associated with this type of intervention?

Common treatments for Age-Related Macular Degeneration (AMD) include anti-VEGF (vascular endothelial growth factor) therapies and corticosteroids, which can be administered via intravitreal or subretinal injections. Known side effects of these treatments include eye pain, increased intraocular pressure, inflammation, and risk of infection. Specific to subretinal administration, as studied in the RGX-314 trial, potential side effects may also include retinal detachment, hemorrhage, and cataract formation. It is crucial for patients to discuss these risks with their ophthalmologist to make an informed decision about their treatment options.

What prior FDA approvals exist?

The FDA has approved several treatments for Age-Related Macular Degeneration (AMD), primarily focusing on anti-VEGF (vascular endothelial growth factor) therapies. These include ranibizumab (Lucentis), aflibercept (Eylea), and brolucizumab (Beovu), which are administered via intravitreal injections. While these treatments are not subretinal like RGX-314, they target similar pathways to inhibit abnormal blood vessel growth and leakage in the retina. Gene therapies like Luxturna (voretigene neparvovec-rzyl) have also been approved for inherited retinal diseases, indicating a growing interest in genetic approaches for retinal conditions.

What other types of research exist?

Promising novel alternatives to RGX-314 for AMD patients include: 1) Faricimab, a bispecific antibody targeting both VEGF-A and Ang-2, which has shown positive results in clinical trials for neovascular AMD. 2) Gene therapy options like ADVM-022, an intravitreal gene therapy delivering aflibercept, which is in advanced clinical trials. 3) Port Delivery System with ranibizumab (PDS), a refillable implant that provides continuous delivery of ranibizumab, reducing the need for frequent injections. These alternatives offer innovative approaches to managing AMD and may be available or in advanced stages of development by 2024.

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Gene Therapy

RGX-314 for Age-Related Macular Degeneration (RGX-314 SRLTFU Trial)

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

Age ≤ 93 years

Diagnosis of bilateral choroidal neovascularization secondary to age-related macular degeneration

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 5 years inclusive of parent study

Awards & highlights

No Placebo-Only Group

Summary

This trial will evaluate the safety & efficacy of RGX-314 over 5yrs in people who have received it in one eye in a previous study. A substudy will look at its safety & immunogenicity in the fellow eye.

Who is the study for?

This trial is for people up to 93 years old with a condition called age-related macular degeneration (AMD) in both eyes. They must have previously received RGX-314 treatment in one eye and be willing to consent. The study excludes those who don't meet the vision criteria or haven't been part of the main observational study.

What is being tested?

The long-term effects and safety of RGX-314 are being studied over five years in patients who've had it before. A subgroup will also get RGX-314 in their other eye, which hasn't been treated yet, to see how well it works there.

What are the potential side effects?

Specific side effects aren't listed here, but generally, treatments like RGX-314 could cause irritation at the injection site, changes in vision, eye discomfort or possible immune reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am 93 years old or younger.

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I have been diagnosed with a specific eye condition due to aging.

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I was in a RGX-314 study and got the treatment once.

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My other eye is affected by wet age-related macular degeneration.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 5 years inclusive of parent study

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~5 years inclusive of parent study

This trial's timeline: 3 weeks for screening, Varies for treatment, and 5 years inclusive of parent study for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Change from baseline in Central Retinal Thickness (CRT) in the study eye

Change from baseline in best corrected visual acuity (BCVA) in the study eye

Fellow eye substudy: Aqueous and Serum RGX-314 transgene product (TP) concentrations in the fellow eye

+6 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

2Treatment groups

Experimental Treatment

Active Control

Group I: RGX-314 Fellow Eye Treatment SubstudyExperimental Treatment1 Intervention

RGX-314 Fellow Eye Treatment

Group II: Main Observational StudyActive Control1 Intervention

All subjects that previously received RGX-314 in a subretinal administration parent study are enrolled into this arm.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

RGX-314

2021

Completed Phase 2

~110

0 / 4Eligibility 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

The most common treatments for Age-Related Macular Degeneration (AMD) include anti-VEGF therapies and gene therapies like RGX-314. Anti-VEGF treatments work by inhibiting the vascular endothelial growth factor, which reduces abnormal blood vessel growth and leakage in the retina, a hallmark of AMD. RGX-314, a gene therapy administered subretinally, aims to provide a long-term solution by enabling retinal cells to produce a therapeutic protein that inhibits VEGF. These treatments are crucial for AMD patients as they help preserve vision and prevent further retinal damage, thereby improving quality of life.