Last updated on February 2019

RGX-314 Gene Therapy for Neovascular AMD Trial


Brief description of study

Excessive vascular endothelial growth factor (VEGF) plays a key part in promoting neovascularization and edema in neovascular (wet) age-related macular degeneration (nAMD). VEGF inhibitors (anti-VEGF), including ranibizumab (LUCENTIS, Genentech) and aflibercept (EYLEA, Regeneron), have been shown to be safe and effective for treating nAMD and have demonstrated improvement in vision. However, anti-VEGF therapy is administered frequently via intravitreal injection and can be a significant burden to the patients. RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein. The long-term, stable delivery of this therapeutic protein following a 1 time gene therapy treatment for nAMD could potentially reduce the treatment burden of currently available therapies while maintaining vision with a favorable benefit:risk profile.

Detailed Study Description

This Phase I, open-label, multiple-cohort, dose-escalation study is designed to evaluate the safety and tolerability of RGX-314 gene therapy in subjects with previously treated nAMD. Four doses will be studied in approximately 24 subjects. Subjects who meet the inclusion/exclusion criteria and have an anatomic response to an initial anti VEGF injection will receive a single dose of RGX-314 administered by subretinal delivery. RGX-314 uses an AAV8 vector that contains a gene that encodes for a monoclonal antibody fragment which binds to and neutralizes VEGF activity. Safety will be the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects will continue to be assessed until 104 weeks following treatment with RGX-314.

Clinical Study Identifier: NCT03066258

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Baltimore location

Baltimore, MD United States
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Boston location

Boston, MA United States
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Philadelphia location 1

Philadelphia, PA United States
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Philadelphia location 2

Philadelphia, PA United States
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Memphis location

Germantown, TN United States
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Houston location

Houston, TX United States
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Santa Barbara location

Santa Barbara, CA United States
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Recruitment Status: Open


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