Safety and Tolerability of RGX-314 (Investigational Product) Gene Therapy for Neovascular AMD Trial
Completed · Phase 1/Phase 2
Conditions studied: Neovascular Age-related Macular Degeneration, Wet Age-related Macular Degeneration
In brief
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.
Key facts
- Study ID
- NCT03066258
- Run by
- REGENXBIO Inc.
- People needed
- 42
- Starts
- 2017-03-29
- Expected to finish
- 2021-06-17
- Last updated by the study team
- 2023-05-16
Who can join
Age: 50 and older, up to 89. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Patients ≥ 50 and ≤ 89 years with a diagnosis of subfoveal CNV (Choroidal neovascularization) secondary to AMD in the study eye receiving prior intravitreal anti-VEGF therapy.
- BCVA (Best Corrected Visual Acuity) between ≤20/63 and ≥20/400 (≤63 and ≥19 Early Treatment Diabetic Retinopathy Study [ETDRS] letters) for the first patient in each cohort followed by BCVA between ≤20/40 and ≥20/400 (≤73 and ≥19 ETDRS letters) for the rest of the cohort.
- History of need for and response to anti-VEGF therapy.
- Response to anti-VEGF at trial entry (assessed by SD-OCT (Spectral Domain Optical Coherence Tomography) at week 1)
- Must be pseudophakic (status post cataract surgery) in the study eye.
- AST (Aspartate aminotransferase)/ALT (Alanine aminotransferase) < 2.5 × ULN (Upper limit of normal); TB (Total bilirubin) < 1.5 × ULN; PT (Prothrombin time) < 1.5 × ULN; Hb > 10 g/dL (males) and > 9 g/dL (females); Platelets > 100 × 10\^3/µL; eGFR (Estimated glomerular filtration rate) > 30 mL/min/1.73 m\^2
- Must be willing and able to provide written, signed informed consent.
You may not qualify if…
- CNV or macular edema in the study eye secondary to any causes other than AMD.
- Any condition preventing visual acuity improvement in the study eye, eg, fibrosis, atrophy, or retinal epithelial tear in the center of the fovea.
- Active or history of retinal detachment in the study eye.
- Advanced glaucoma in the study eye.
- 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.
- Presence of an implant in the study eye at screening (excluding intraocular lens).
- Myocardial infarction, cerebrovascular accident, or transient ischemic attacks within the past 6 months.
- Uncontrolled hypertension (systolic blood pressure [BP] >180 mmHg, diastolic BP >100 mmHg) despite maximal medical treatment.
Where it is running
- Santa Barbara location — Santa Barbara, California, United States
- Baltimore location — Baltimore, Maryland, United States
- Boston location — Boston, Massachusetts, United States
- Reno location — Reno, Nevada, United States
- Philadelphia location 1 — Philadelphia, Pennsylvania, United States
- Philadelphia location 2 — Philadelphia, Pennsylvania, United States
- Memphis location — Germantown, Tennessee, United States
- Houston location — Houston, Texas, United States
Full record on ClinicalTrials.gov
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