Pivotal 2 Study of RGX-314 Gene Therapy in Participants With nAMD

What this study is about

ABBV-RGX-314 (also known as RGX-314 and surabgene lomparvovec (sura-vec)) is being developed as a novel one-time gene therapy for the treatment of neovascular (wet) age-related macular degeneration (wet AMD). 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 (VEGF) therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients. These therapies, however, require life-long intraocular injections, typically repeated every four to 12 weeks in frequency, to maintain efficacy. Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time. ABBV-RGX-314 is being developed as a potential one-time treatment for wet AMD.

Who can join (things the study team will check)

✅ You may be able to join if…

• Age ≥ 50 years and ≤ 89 years
• An ETDRS BCVA letter score between ≤ 78 and ≥ 40 in the study eye
• Diagnosis of subfoveal choroidal neovascularization (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

✅ You may be able to join if…

• 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 be able to join if…

• CNV or macular edema in the study eye secondary to any causes other than AMD
• Subfoveal fibrosis or atrophy in the study eye
• Any condition in the investigator's opinion that could limit VA improvement in the study eye
• Advanced glaucoma or history of secondary glaucoma in the study eye
• Myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months
• 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 medicinal product, other than an intravitreal therapy for AMD, in the 6 months prior to Week -6
• Prior treatment with gene therapy

🚫 You may not be able to join if…

• 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
• 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
• History of intravitreal therapy in the study eye, such as intravitreal steroid injection or investigational medicinal product, other than an intravitreal therapy for AMD, in the 6 months prior to Week -6.
• Prior treatment with gene therapy (*) For previously treated crossover participants, criteria apply to the eye not treated in the main study only. Note: Other inclusion/exclusion criteria apply

Where this trial is running

• Barnet Dulaney Eye Center-Phoenix /ID# 256340, Phoenix, Arizona, United States
• Retinal Research Institute /ID# 256238, Phoenix, Arizona, United States
• Retina Macula Institute of Arizona /ID# 271026, Scottsdale, Arizona, United States
• California Retina Consultants - Bakersfield /ID# 256240, Bakersfield, California, United States
• Retina Vitreous Assoc Med Grp /ID# 256246, Beverly Hills, California, United States
• Retinal Diagnostic Center /ID# 256262, Campbell, California, United States
• The Retina Partners - Encino /ID# 259660, Encino, California, United States
• Retina Consultants of Orange County /ID# 256267, Fullerton, California, United States
• Jacobs Retina Center at UCSD/ID# 256320, La Jolla, California, United States
• California Retina Consultants - Oxnard - North Ventura Road /ID# 262883, Oxnard, California, United States
• Byers Eye Institute Stanford /ID# 262853, Palo Alto, California, United States
• Retina Consultants of San Diego /ID# 256258, Poway, California, United States

+ 169 more sites.

Who to contact

Patient Advocacy · 1-833-711-0349 · Patientadvocacy@regenxbio.com

It's completely normal to call and ask questions before deciding anything. Mention the study ID: NCT05407636.