RGX-314 for Age-related Macular Degeneration

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Age-related Macular Degeneration+5 MoreRGX-314 - Genetic
Eligibility
50 - 89
All Sexes
What conditions do you have?
Select

Study Summary

This trial is studying a new gene therapy called RGX-314 for wet age-related macular degeneration. Wet AMD is when new, leaky blood vessels form in the retina and cause vision loss. Current treatments require life-long intraocular injections, typically repeated every four to twelve weeks, to maintain efficacy. RGX-314 is being developed as a potential one-time treatment for wet AMD.

Eligible Conditions
  • Convergence Insufficiency
  • Age-related Macular Degeneration
  • Neovascular Age-related Macular Degeneration
  • Wet Age-Related Macular Degeneration
  • Wet Age-related Macular Degeneration
  • Age-related Macular Degeneration (AMD)

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

1 Primary · 6 Secondary · Reporting Duration: At Week 14, Week 26, Week 38, Week 54, and Week 98

Week 98
Aqueous RGX-314 target protein (TP) concentrations
At Week 54
Mean change from baseline in Best Corrected Visual Acuity (BCVA)
Week 98
Mean change from baseline in Central Retinal Thickness (CRT) and Center Point Thickness (CPT) as measured by Spectral Domain Optical Coherence Tomography (SD-OCT)
Week 98
Mean change from baseline in BCVA
Week 98
Mean reduction in supplemental anti VEGF injection annualized rate compared with the prior 52 weeks preceding the first ranibizumab injection received as part of the Active Run-in Period (RGX 314 randomized participants)
Mean supplemental anti-VEGF injection annualized rate in the RGX-314 arms
Through Week 98
Incidences of ocular and overall adverse events (AEs)

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Trial Design

3 Treatment Groups

Control Arm
1 of 3
RGX-314 Dose 1
1 of 3
RGX-314 Dose 2
1 of 3

Active Control

Experimental Treatment

300 Total Participants · 3 Treatment Groups

Primary Treatment: RGX-314 · No Placebo Group · Phase 2 & 3

RGX-314 Dose 1
Genetic
Experimental Group · 1 Intervention: RGX-314 · Intervention Types: Genetic
RGX-314 Dose 2
Genetic
Experimental Group · 1 Intervention: RGX-314 · Intervention Types: Genetic
Control Arm
Biological
ActiveComparator Group · 1 Intervention: Ranibizumab (LUCENTIS®) · Intervention Types: Biological

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: at week 14, week 26, week 38, week 54, and week 98

Who is running the clinical trial?

REGENXBIO, Inc.Lead Sponsor
19 Previous Clinical Trials
1,110 Total Patients Enrolled
Regenxbio Inc.Lead Sponsor
19 Previous Clinical Trials
1,110 Total Patients Enrolled
REGENXBIO Inc.Lead Sponsor
21 Previous Clinical Trials
1,328 Total Patients Enrolled

Eligibility Criteria

Age 50 - 89 · All Participants · 6 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You are willing and able to provide written, signed informed consent for this study.
You must have demonstrated a meaningful response to anti-VEGF therapy at study entry.

Who else is applying?

What state do they live in?
Florida33.3%
Mississippi33.3%
Missouri33.3%
How old are they?
65+100.0%
What site did they apply to?
Germantown Location100.0%
What portion of applicants met pre-screening criteria?
Did not meet criteria66.7%
Met criteria33.3%
How many prior treatments have patients received?
3+100.0%