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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
Must be pseudophakic (at least 12 weeks postcataract surgery) in the study eye
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at week 24 and 48
Awards & highlights

Study Summary

This trialis testing a gene therapy to treat Wet AMD, which causes vision loss due to new, leaky blood vessels in the retina. It will compare two different doses of the therapy in humans.

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.Check my eligibility
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.See study design
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.
Select...
I had cataract surgery in my study eye more than 12 weeks ago.
Select...
I am between 50 and 89 years old.
Select...
I responded well to previous anti-VEGF therapy.

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
RGX-314 target protein concentration in aqueous humor
Secondary outcome measures
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%
Photopsia
33%
Conjunctival Haemorrhage
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

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
2017
Completed Phase 2
~50

Find a Location

Who is running the clinical trial?

Regenxbio Inc.Lead Sponsor
19 Previous Clinical Trials
2,233 Total Patients Enrolled
REGENXBIO, Inc.Lead Sponsor
19 Previous Clinical Trials
2,233 Total Patients Enrolled
REGENXBIO Inc.Lead Sponsor
23 Previous Clinical Trials
2,475 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are the parameters of this research experiment compatible with my qualifications?

"This clinical trial seeks 60 individuals aged 50 to 89 with wet macular degeneration. Eligibility is limited to males and females who possess a Early Treatment Diabetic Retinopathy Study (ETDRS) BCVA-letter score of 40 or higher and 78 or lower in the study eye at Screening, have been previously treated with anti-VEGF agents, attained meaningful response to these interventions, are pseudophakic for 12 weeks minimum in the study eye, and agree via signed informed consent that they will participate."

Answered by AI

Are the elderly allowed to enter this research study?

"The age range for inclusion in this medical trial is 50 to 89. There are 10 trials specifically designed for those below 18, and 153 clinical studies tailored towards the over 65 demographic."

Answered by AI

Is recruitment currently being conducted for this research endeavor?

"According to the information sourced from clinicaltrials.gov, this medical trial is currently recruiting new participants. The initial posting of this study was on February 22nd 2021 and it has been updated most recently on November 21st 2022."

Answered by AI

What is the maximum enrolment for this research project?

"That is correct. According to clinicaltrials.gov, this research study began recruiting on February 22nd 2021 and its most recent update was made November 21st 2022; with the aim of enrolling 60 participants from 26 varying sites."

Answered by AI

Has RGX-314 received clearance from the FDA?

"Due to the Phase 2 status of RGX-314, there is some evidence for safety yet no confirmation regarding efficacy; thus, our team at Power assigned it a rating of 2."

Answered by AI

Are there many sites in America hosting this trial?

"Patients can enroll in this clinical research study at Ophthalmic Consultants of Boston (Boston, MA), Sierra Eye Associates (Reno, NV) and Charles Retina Institute (Germantown, TN). An additional 26 sites are also participating."

Answered by AI
~15 spots leftby Mar 2025