RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Diabetic Retinopathy (DR) With and Without Center Involved-Diabetic Macular Edema (CI-DME)

NCT04567550 · tracked via the Priya Life Science USA tracker

Sponsor

AbbVie

Phase

Phase 2

Started

2020-11-20

Last updated

2026-08-19

Condition(s) studied

Diabetic Retinopathy (DR)Center-Involved Diabetic Macular Edema (CI-DME)

Investigational drug(s) / intervention(s)

ABBV-RGX-314 Dose 1ABBV-RGX-314 Dose 2ABBV-RGX-314 Dose 3Topical Steroid →ABBV-RGX-314 Dose 4Aflibercept →

ABBV-RGX-314 Dose 1: AAV8 vector containing a transgene for anti-VEGF fab (Dose 1)

ABBV-RGX-314 Dose 2: AAV8 vector containing a transgene for anti-VEGF fab (Dose 2)

ABBV-RGX-314 Dose 3: AAV8 vector containing a transgene for anti-VEGF fab (Dose 3)

Topical Steroid: Topical Steroid

ABBV-RGX-314 Dose 4: AAV8 vector containing a transgene for anti-VEGF fab (Dose 4)

Aflibercept: Aflibercept

Study summary

ABBV-RGX-314 is being developed as a novel, potential one-time gene therapy treatment for the treatment of Diabetic Retinopathy (DR) with and without Center-Involved Diabetic Macular Edema (CI-DME). DR is a chronic and progressive complication of diabetes mellitus. It is a sight-threatening disease characterized in the early stages by neuronal and vascular dysfunction in the retina, and later by neovascularization that leads to further deterioration of functional vision. Despite the availability of current treatments, diabetic retinopathy remains the leading cause of vision loss in working-age adults, those between the ages of 20 and 74. Existing treatment with anti-VEGF agents, although shown to be effective, are limited by short therapeutic half-lives, which then require frequent intravitreal injections over the patient's lifetime, resulting in increased risk of associated adverse events and significant treatment burden. Due to the burden of treatment, patients often do not closely adhere to treatment regimens and experience sub-optimal outcomes and a decline in vision.

Eligibility

Sex

ALL

Min age

25 Years

Max age

89 Years

Healthy volunteers

Part 1 (DR without CI-DME): Inclusion Criteria: * Patients 25-89 years of age with a diabetic retinopathy (DR) diagnosis of nonproliferative diabetic retinopathy (NPDR) and proliferative diabetic retinopathy (PDR) secondary to diabetes mellitus Type 1 or 2 for which PRP or anti-VEGF injections can be safely deferred for at least 6 months * HbA1c \< 12%. * Best corrected visual acuity (BCVA) Early Treatment Diabetic Retinopathy Study (ETDRS) letter score in the study eye of ≥69 letters (approximate Snellen equivalent of 20/40 or better). * Prior history of CI-DME in the study eye is acceptable. * Must be willing and able to provide written, signed informed consent. Exclusion Criteria: * Neovascularization in the study eye from a cause other than DR. * Presence of any active CI-DME. * Active or history of retinal detachment in the study eye. * Any evidence or documented history of PRP or retinal laser in the study eye. * Patients who had a prior vitrectomy surgery. * Women of childbearing potential. Part 2 (DR with CI-DME): Inclusion Criteria: * Patients 25-89 years of age with diabetic retinopathy secondary to diabetes mellitus Type 1 or 2. * HbA1c \< 12% * Macular thickening secondary to DME involving the center of the fovea, CST on SD-OCT (≥ 325 μm) * Best corrected visual acuity (BCVA) Early Treatment Diabetic Retinopathy Study (ETDRS) letter score in the study eye of 78-25 letters (approximate Snellen equivalent of 20/32 to 20/320) * Participants must have demonstrated a meaningful response to anti-VEGF therapy. * Must be willing and able to provide written, signed informed consent Exclusion Criteria: * Neovascularization in the study eye from a cause other than DR. * Active or history of retinal detachment in the study eye. * Any evidence or documented history of PRP or retinal laser in the study eye. * Patients who had a prior vitrectomy surgery. * Women of childbearing potential. Note: Other inclusions/exclusions criteria apply.

Primary outcome measure(s)

Part 1: Proportion of participants achieving a 2-step or greater improvement in DR in the study eye per the ETDRS-DRSS on 4 widefield digital stereoscopic fundus photography at Week 48 — At Week 48
To evaluate the effect of ABBV-RGX-314 on DR by the ETDRS DRSS at Week 48.
Part 2: Mean change from baseline in Best Corrected Visual Acuity (BCVA) in the study eye at Week 54. — At Week 54
To evaluate the effect of ABBV-RGX-314 on BCVA at Week 54.

Trial sites (25)

Facility	City	Region
Retinal Research Institute, LLC	Phoenix	Arizona
Barnet Dulaney Perkins Eye Center	Phoenix	Arizona
California Retina Consultants	Bakersfield	California
Retina-Vitreous Associates Medical Group	Beverly Hills	California
Retinal Diagnostic Center	Campbell	California
Northern California Retina Vitreous Associates Medical Group, Inc.	Mountain View	California
California Eye Specialists Medical Group, Inc	Pasadena	California
Retinal Consultants San Diego	Poway	California
California Retina Consultants	Santa Barbara	California
Southeast Retina Center, PC	Augusta	Georgia
University Retina and Macula Associates, PC	Oak Forest	Illinois
Springfield Clinic	Springfield	Illinois
Wilmer Eye Institute/Johns Hopkins University School of Medicine	Baltimore	Maryland
Cumberland Valley Retina Consultants	Hagerstown	Maryland
Ophthalmic Consultants of Boston	Boston	Massachusetts
Sierra Eye Associates	Reno	Nevada
NJ Retina	Teaneck	New Jersey
Vision Research Center Eye Associates of New Mexico	Albuquerque	New Mexico
Duke University Eye Center	Durham	North Carolina
Mid Atlantic Retina	Philadelphia	Pennsylvania
Charles Retina Institute, P.C.	Germantown	Tennessee
Retina Research Institute of Texas, LLC	Abilene	Texas
Austin Clinical Research, LLC	Austin	Texas
Star Retina	Burleson	Texas
Retinal Consultants of Texas	The Woodlands	Texas

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04567550 on ClinicalTrials.gov ↗ ← All trials in the USA