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

Contact Investigators or Research Sites near you

Start Over

Baltimore location

Baltimore, MD United States
  Connect »

Boston location

Boston, MA United States
  Connect »

Philadelphia location 1

Philadelphia, PA United States
  Connect »

Philadelphia location 2

Philadelphia, PA United States
  Connect »

Memphis location

Germantown, TN United States
  Connect »

Houston location

Houston, TX United States
  Connect »

Santa Barbara location

Santa Barbara, CA United States
  Connect »

Recruitment Status: Open

Brief Description Eligibility Contact Research Team

Receive Emails About New Clinical Trials!

Sign up for our FREE service to receive email notifications when clinical trials are posted in the medical category of interest to you.