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
- Biological: RGX-314
Regenxbio Inc. -- RGX-314-001
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.
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)
|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|