Pivotal 1 Study of ABBV-RGX-314 (Also Known as RGX-314) Gene Therapy Administered Via Subretinal … (NCT04704921) | Clinical Trial Compass
Active — Not RecruitingPhase 2/3
Pivotal 1 Study of ABBV-RGX-314 (Also Known as RGX-314) Gene Therapy Administered Via Subretinal Delivery One Time in Participants With nAMD
United States671 participantsStarted 2020-12-29
Plain-language summary
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.
Who can participate
Age range
50 Years – 89 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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
. An ETDRS BCVA letter score between ≤ 83 and ≥ 40 in both eyes
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Mean change from baseline in Best Corrected Visual Acuity (BCVA)
Timeframe: At Week 54
2
Bilateral Treatment Substudy: Incidence of ocular AEs and any SAEs
. Diagnosis of subfoveal choroidal neovascularization (CNV) secondary to AMD in both eyes
Exclusion criteria
. 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