Pivotal 1 Study of ABBV-RGX-314 (Also Known as RGX-314) Gene Therapy Administered Via Subretinal Delivery One Time in Participants With nAMD

Running, not enrolling · Phase 2/Phase 3

Conditions studied: AMD, nAMD, Wet Age-related Macular Degeneration, wAMD, Wet AMD, CNV, Neovascular AMD, Neovascular Age-related Macular Degeneration, Choroidal Neovascularization

In brief

ABBV-RGX-314 (also known as RGX-314) is being developed as a novel one-time gene therapy for the treatment of neovascular (wet) age-related macular degeneration (wet AMD or nAMD). Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-vascular endothelial growth factor (anti-VEGF) therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to maintain or prevent progression of vision loss in the majority of patients. These therapies, however, require life-long intraocular injections, typically repeated every 4 to 16 weeks in frequency, to maintain efficacy. Due to the burden of these treatments, patients often experience a decline in vision with reduced frequency of treatment over time.

Key facts

Study ID

NCT04704921

Run by

AbbVie

People needed

671

Starts

2020-12-29

Expected to finish

2027-11-01

Last updated by the study team

2026-04-28

Who can join

Age: 50 and older, up to 89. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Age ≥ 50 years and ≤ 89 years
• An ETDRS BCVA letter score between ≤ 78 and ≥ 40 in the study eye
• Diagnosis of subfoveal CNV secondary to AMD in the study eye previously treated with anti-VEGF
• Must be pseudophakic (at least 12 weeks postcataract surgery) in the study eye.
• Willing and able to provide written, signed informed consent for this study
• Participants must have demonstrated a meaningful response to anti-VEGF therapy at study entry
• Inclusion Criteria (Bilateral Treatment Substudy)*:
• An ETDRS BCVA letter score between ≤ 83 and ≥ 40 in both eyes
• Diagnosis of subfoveal choroidal neovascularization (CNV) secondary to AMD in both eyes
• Must be pseudophakic (at least 12 weeks postcataract surgery) in both eyes
• Willing and able to provide written, signed informed consent for this study
• Newcomers must have active disease in the study eye; crossover participants must have active disease in the eye not treated in the main study

You may not qualify if…

• CNV or macular edema in the study eye secondary to any causes other than AMD
• Subfoveal fibrosis or atrophy in the study eye, as determined by CRC
• Any condition in the investigator's opinion that could limit VA improvement in the study eye
• Active or history of retinal detachment, or current retinal tear that cannot be treated, in the study eye
• Advanced glaucoma or history of secondary glaucoma in the study eye
• History of intraocular surgery in the study eye within 12 weeks prior to randomization
• History of intravitreal therapy in the study eye, such as intravitreal steroid injection or investigational product, other than anti-VEGF therapy, in the 6 months prior to Screening Visit 1
• Prior treatment with gene therapy
• Recent myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months
• Exclusion Criteria (Bilateral Treatment Substudy)*:
• CNV or macular edema in either eye secondary to any causes other than AMD
• Subfoveal fibrosis or atrophy in either eye
• Any condition in the investigator's opinion that could limit VA improvement in either eye
• Active or history of retinal detachment, or current retinal tear that cannot be treated in either eye
• Advanced glaucoma or history of secondary glaucoma in either eye
• Myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months
• History of intraocular surgery in either eye within 12 weeks prior to randomization (Week -2)
• History of intravitreal therapy in either eye, such as intravitreal steroid injection or investigational product, other than anti-VEGF therapy, in the 6 months prior to screening
• Prior treatment with gene therapy (*) For previously treated crossover participants, criteria apply to the eye not treated in the main study only.

Where it is running

• Barnet Dulaney Perkins Eye Center - Sun City /ID# 256055 — Sun City, Arizona, United States
• University of Arkansas for Medical Sciences /ID# 271290 — Little Rock, Arkansas, United States
• Retina Vitreous Assoc Med Grp /ID# 256299 — Beverly Hills, California, United States
• Retinal Diagnostic Center /ID# 256137 — Campbell, California, United States
• The Retina Partners - Encino /ID# 256054 — Encino, California, United States
• Retina Consultants of Orange County /ID# 256152 — Fullerton, California, United States
• Salehi Retina Institute /ID# 263485 — Huntington Beach, California, United States
• UC Irvine/Gavin Herbert Eye Institute /ID# 256145 — Irvine, California, United States
• Northern California Retina Vitreous Associates Medical Group, Inc /ID# 256298 — Mountain View, California, United States
• UCLA Doheny Eye Center /ID# 256120 — Pasadena, California, United States
• California Eye Specialists Medical Group Inc. /ID# 256079 — Pasadena, California, United States
• Retina Consultants of San Diego /ID# 256021 — Poway, California, United States
• Retinal Consultants Medical Group /ID# 256047 — Sacramento, California, United States
• West Coast Retina /ID# 256448 — San Francisco, California, United States
• University of California, San Francisco /ID# 256130 — San Francisco, California, United States
• Orange County Retina Medical Group /ID# 256073 — Santa Ana, California, United States
• California Retina Consultants - Santa Barbara /ID# 256017 — Santa Barbara, California, United States
• Retina Consultants of Southern Colorado /ID# 256069 — Colorado Springs, Colorado, United States
• Southwest Retina Research Center /ID# 256136 — Durango, Colorado, United States
• Colorado Retina Associates /ID# 256121 — Lakewood, Colorado, United States
• Retina Group of New England - Waterford /ID# 256071 — Waterford, Connecticut, United States
• Advanced Research, LLC /ID# 275451 — Deerfield Beach, Florida, United States
• Vitreoretinal Associates, P.A. /ID# 256150 — Gainesville, Florida, United States
• Florida Retina Consultants /ID# 265661 — Lakeland, Florida, United States
• Retinal Research Institute /ID# 256019 — Phoenix, Arizona, United States

Full record on ClinicalTrials.gov

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