RGX-314 Gene Therapy for Neovascular AMD Trial


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.

Study Start Date

March, 31 2017

Estimated Completion Date

February 2020


  • Biological: RGX-314

Study ID

Regenxbio Inc. -- RGX-314-001



Trial ID


Study Type


Trial Phase

Phase 1

Enrollment Quota



Regenxbio Inc.

Inclusion Criteria

    1. Patients ? 50 years with a diagnosis of subfoveal CNV secondary to AMD in the study eye receiving prior intravitreal anti-VEGF therapy. 2. BCVA between ?20/100 and ?20/400 (?53 and ?19 Early Treatment Diabetic Retinopathy Study [ETDRS] letters) for the first patient in each cohort followed by BCVA between ?20/63 and ?20/400 (?63 and ?19 ETDRS letters) for the rest of the cohort. 3. History of need for and response to anti-VEGF.therapy. 4. Response to anti-VEGF at trial entry (assessed by SD-OCT at week 1) 5. Must be pseudophakic (status post cataract surgery) in the study eye. 6. AST/ALT < 2.5 × ULN TB < 1.5 × ULN PT < 1.5 × ULN Hb > 10 g/dL (males) and > 9 g/dL (females) Platelets > 100 × 10^3/µL eGFR > 30 mL/min/1.73 m^2 7. Must be willing and able to provide written, signed informed consent.

Exclusion Criteria

    1. CNV or macular edema in the study eye secondary to any causes other than AMD. 2. Any condition preventing visual acuity improvement in the study eye, eg, fibrosis, atrophy, or retinal epithelial tear in the center of the fovea. 3. Active or history of retinal detachment in the study eye. 4. Advanced glaucoma in the study eye. 5. 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. 6. Presence of an implant in the study eye at screening (excluding intraocular lens). 7. Myocardial infarction, cerebrovascular accident, or transient ischemic attacks within the past 6 months. 8. Uncontrolled hypertension (systolic blood pressure [BP] >180 mmHg, diastolic BP >100 mmHg) despite maximal medical treatment.




50 Years and older

Accepts Healthy Volunteers


Study Locations and Contact Information (6)

Study Location Distance Name Phone Email
Boston location - Boston, Massachusetts 2.8 miles None None None
Philadelphia location 2 - Philadelphia, Pennsylvania 269.5 miles None None None
Philadelphia location 1 - Philadelphia, Pennsylvania 270.8 miles None None None
Baltimore location - Baltimore, Maryland 356.0 miles None None None
Memphis location - Germantown, Tennessee 1,131.3 miles None None None
Houston location - Houston, Texas 1,609.9 miles None None None

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