A Phase 2 interventional study of RGX-314 in Neovascular Age-related Macular Degeneration, Wet Macular Degeneration and Wet Age-related Macular Degeneration, sponsored by AbbVie. Completed at 13 sites in United States. Open to participants aged 50 Years to 89 Years. Per ClinicalTrials.gov, last updated 2024-09-05.
Sponsored by AbbVie · Phase 2, Interventional, and Treatment
RGX-314 is being developed as a novel one-time gene therapy for the treatment of neovascular (wet) age-related macular degeneration (nAMD) also referred to as Wet AMD. Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. The purpose of this phase 2, open label study is to evaluate whether different doses of RGX-314 from two different formulations (clinical versus eventual commercial formulation) perform the same in humans when delivered by subretinal administration
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-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 repeated intraocular injections to maintain efficacy. Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time. RGX-314 is being developed as a potential one-time treatment for wet AMD. RGX-314 uses an AAV8 vector that contains a gene that encodes for a monoclonal antibody fragment which binds to and neutralizes VEGF activity. This phase 2, open label study will explore the pharmacodynamics of two doses in two formulations of RGX-314 gene therapy via subretinal delivery in patients with neovascular Age-related Macular Degeneration. Approximately 60 patients (15 per cohort) who meet the inclusion/exclusion criteria will be enrolled in 4 sequential dose cohorts. A dose cohort will be comprised of 1 of 2 doses of RGX-314 in 1 of 2 formulations in order to explore the pharmacodynamics of RGX-314 based on aqueous humor transgene product (TP) concentrations. If the participants meet the study criteria and choose to participate in the study, their participation in the study will last about 12 months.
1,474 studies on the registry are indexed under Macular Degeneration; 206 are open to participants now.
This study's enrollment of 60 is above the median of 51 across 985 interventional studies indexed under Macular Degeneration.
Browse Macular Degeneration studies →AbbVie is the lead sponsor of 953 studies on the registry; 136 are open to participants now.
Of its 350 completed or terminated interventional studies of FDA-regulated products, 210 (60%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Dose 1 of RGX-314
Genetic: RGX-314
Dose 1 of RGX-314
Genetic: RGX-314
Dose 2 of RGX-314
Genetic: RGX-314
Dose 2 of RGX-314
Genetic: RGX-314
RGX-314 is a recombinant adeno-associated virus gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein
RGX-314 target protein concentration in aqueous humor
Time frame: At Week 24
Incidence and severity of ocular Adverse Events (AEs) and overall AEs
Evaluate the safety and tolerability of RGX-314
Time frame: Through Week 48
Changes in Best Corrected Visual Acuity (BCVA)
BCVA measured by Early Treatment Diabetic Retinopathy Study (ETDRS)
Time frame: At Week 24 and 48
Changes in Central Retinal Thickness (CRT)
CRT is measured by spectral domain optical coherence tomography (SD-OCT)
Time frame: At Week 24 and 48
Mean Supplemental anti-VEGF injection annualized rate through week 24 and week 48
To assess the need for supplemental anti-VEGF therapy over 48 weeks
Time frame: Through Week 24 and week 48
Plan to share: No
No publications or documents are linked to this record.
This study is completed, as verified in Sep 2024. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
AbbVie